Page last updated: 2024-08-23

1-deoxynojirimycin and miglustat

1-deoxynojirimycin has been researched along with miglustat in 338 studies

Research

Studies (338)

Timeframe	Studies, this research(%)	All Research%
pre-1990	2 (0.59)	18.7374
1990's	35 (10.36)	18.2507
2000's	123 (36.39)	29.6817
2010's	150 (44.38)	24.3611
2020's	28 (8.28)	2.80

Authors

Authors	Studies
Asano, N; Baba, M; Kizu, H; Matsui, K; Okamoto, M; Oseki, K; Tomioka, E	1
Asano, N; Compain, P; Godin, G; Ikeda, K; Martin, OR; Yu, L	1
Adachi, I; Asano, N; Compain, P; Godin, G; Ikeda, K; Kato, A; Martin, O; Yu, L	1
Blériot, Y; Davies, GJ; Dumon, C; Gilbert, HJ; Gratien, J; Heck, MP; Morland, C; Offen, WA; Smith, NL; Stick, RV; Tailford, LE; Vasella, A	1
Aerts, JM; Donker-Koopman, WE; Overhand, M; Overkleeft, HS; Risseeuw, MD; van den Berg, RJ; van der Marel, GA	1
Aerts, JM; Bijl, N; Boot, RG; Copeland, D; Ghauharali, K; Groen, AK; Groener, JE; Liu, H; Meijer, AJ; O'Shea, TJ; Ottenhoff, R; Overkleeft, HS; Song, H; van den Berg, RJ; van der Marel, GA; van Eijk, M; Wennekes, T; Yew, N	1
Codd, R; Liao, V; Lifa, T; Liu, J; Obando, D	1
Alonzi, DS; Butters, TD; Cumpstey, I; Eszter Borbas, K; Ramstadius, C	1
Adachi, I; Hayashi, E; Hirono, S; Imahori, T; Kato, A; Koseki, J; Miyauchi, S; Nakagome, I; Nash, RJ; Natori, Y; Shimaoka, H; Takahata, H; Yoshimura, Y	1
Benjamin, ER; Boyd, RE; Khanna, R; Lee, G; Rybczynski, P; Valenzano, KJ; Wustman, BA	1
Block, TM; Chang, J; Du, Y; Guo, F; Guo, J; Guo, JT; Kinney, W; Lam, P; Li, Y; Lu, H; Ma, J; Su, Q; Wu, S; Yan, Z; Yang, K; Zhang, X	1
Fleet, GWJ; Jia, YM; Kanekiyo, U; Kato, A; Kishida, M; Li, YX; Lu, TT; Nakagome, I; Nash, RJ; Shinzawa, K; Tanaka, N; Yoshida, T; Yoshimura, K; Yu, CY	1
Fleet, GWJ; Jia, YM; Kato, A; Kise, M; Li, YX; Shimadate, Y; Wang, JZ; Yu, CY	1
Jacob, GS; Karlsson, GB; Platt, FM	1
Balwierz, PS; Gad, SC; Guzzie, PJ; Oshiro, Y; Rohrbacher, E; Soelter, SG	1
Bowlin, TL; Kang, MS; Liu, PS; Sunkara, PS; Taylor, DL; Tyms, AS	1
Dedera, D; Ratner, L; vander Heyden, N	2
Fujimoto, K; Kornfeld, R	1
Jacob, GS; Jones, IM	1
Block, T; Butters, T; Dwek, R; Lu, X; Mehta, A	1
Cook, CS; Karabatsos, PJ; Karim, A; Schoenhard, GL	1
Barclay, AN; Bodian, DL; Butters, TD; Daenke, S; Davies, EA; Davis, SJ; Dwek, RA; Jones, EY; Stuart, DI; van der Merwe, PA	1
Butters, TD; Collin, M; Davis, SJ; Dwek, RA; Fischer, PB; Gordon, S; James, W; Karlsson, GB; Platt, FM	1
Aspinall, RL; Collier, AC; Coombs, R; Fass, RJ; Fife, KH; Fischl, MA; Kremer, AB; Pottage, JC; Powderly, WG; Resnick, L	1
Butters, TD; Dwek, RA; Karlsson, GB; Neises, GR; Platt, FM	1
Butters, TD; Dwek, RA; Neises, GR; Platt, FM	1
Block, TM; Blumberg, BS; Dwek, RA; Foster, GR; Gerlich, WH; Lu, X; Platt, FM	1
Ratner, L; Vander Heyden, N	1
Butters, TD; Dwek, RA; Karlsson, GB; Platt, FM	1
Fischl, M; Karim, A; Kessler, H; Merigan, T; Pottage, J; Powderly, W; Richman, D; Sherman, J; Smith, S; Tierney, M	1
Abe, A; Radin, NS; Shayman, JA	1
Block, TM; Gerlich, WH; Lu, X	1
Butters, TD; Dwek, RA; Fischer, PB; Karlsson, GB; Platt, FM	1
Dwek, RA; Fischer, PB; Karlsson, GB; Platt, FM	1
Butters, TD; Dwek, RA; Neises, GR; Perry, VH; Platt, FM; Proia, RL; Reinkensmeier, G; Townsend, MJ; Winchester, B	1
Dwek, RA; Petrescu, AJ; Petrescu, SM; Platt, FM; Titu, HN	1
Dwek, RA; Harvey, DJ; Hunter, AP; Küster, B; Wheeler, SF	1
Butters, TD; Dwek, RA; Platt, FM; Reinkensmeier, G	1
Butters, TD; Dwek, RA; Petrescu, AJ; Petrescu, S; Platt, FM; Reinkensmeier, G; Wormald, MR	1
Butters, TD; Neises, GR; Ornberg, RL; Platt, FM; Woodman, PG	1
Chavany, C; Jendoubi, M	1
Bieberich, E; Freischütz, B; Suzuki, M; Yu, RK	1
Butters, TD; Cortina-Borja, M; Dwek, RA; Hunnam, V; Jeyakumar, M; Perry, VH; Platt, FM; Proia, RL	1
Butters, TD; Davis, SJ; Harlos, K; Ikemizu, S; Jones, EY; Sparks, LM; Stuart, DI	1
Branza-Nichita, N; Dwek, RA; Negroiu, G; Petrescu, AJ; Petrescu, SM	1
Marcus, NY; Perlmutter, DH	1
Branza-Nichita, N; Costin, GE; Dwek, RA; Negroiu, G; Petrescu, AJ; Petrescu, SM; Titu, H	1
Abe, A; Arend, LJ; Brady, RO; Lee, L; Lingwood, C; Shayman, JA	1
Eto, S; Fukuda, MN; Misago, M; Tsukada, J	1
Andersson, U; Butters, TD; Dwek, RA; Platt, FM	1
Aerts, J; Butters, T; Cox, T; Dwek, R; Elstein, D; Gow, I; Hollak, C; Hrebícek, M; Lachmann, R; Moyses, C; Platt, F; van Weely, S; Zimran, A	1
Vellodi, A; Winchester, B; Young, E	1
Abe, A; Brady, RO; Gregory, S; Killen, PD; Kulkarni, A; Lee, L; Shayman, JA	1
Chan-Fook, C; Clarke, BE; Jiang, WR; Jones, IM; Maidens, C; McKeating, JA; Zitzmann, N	1
Dwek, RA; Negroiu, G; Petrescu, SM	1
Adam, A; Butters, TD; Dwek, RA; Mellor, HR; Platt, FM	1
Mistry, PK	1
Kranda, M	1
Barranger, JA	1
Butters, TD; Cortina-Borja, M; Dwek, RA; Jeyakumar, M; Norflus, F; Perry, VH; Platt, FM; Proia, RL; Tifft, CJ	1
Proia, RL; Tifft, CJ	1
Butters, TD; Dwek, RA; Platt, FM; Priestman, DA	1
Lachmann, RH; Platt, FM	1
Branza-Nichita, N; Carrouée-Durantel, S; Durantel, D; Dwek, RA; Zitzmann, N	1
El-Abbadi, M; Manfredi, MG; Mukherjee, P; Platt, FM; Ranes, MK; Seyfried, TN	1
Somers, KL; Thrall, MA; Walkley, SU; Zervas, M	1
Block, TM; Conyers, B; Dwek, RA; Jordan, R; Mehta, A; Nikolaeva, OV; Wang, L	1
Costin, GE; Dwek, RA; Nichita, N; Petrescu, SM; Trif, M	1
Block, TM; Dwek, RA; Jordan, R; Mehta, A; Ouzounov, S	1
Cresswell, P; Kang, SJ	1
Butters, TD; Dwek, RA; Jeyakumar, M; Platt, FM	1
Butters, TD; Charlton, HM; Dwek, RA; Jeyakumar, M; Moore, HD; Platt, FM; van der Spoel, AC	1
Butters, T; Dwek, R; Frendeus, B; Platt, F; Svanborg, C; Svensson, M	1
Butters, TD; d'Azzo, A; Dwek, RA; Elliot-Smith, E; Jeyakumar, M; Perry, VH; Platt, FM; Smith, DA; Thomas, R; van der Spoel, AC	1
Cooper, T	1
Futerman, AH; Jeyakumar, M; Lloyd-Evans, E; Pelled, D; Platt, FM; Riebeling, C	1
Albrecht, RF; Balyasnikova, IV; Chemodanova, EE; Danilov, SM; Kost, OA; Nikolskaya, II	1
Andersson, U; Butters, TD; Dwek, RA; Heare, T; Jeyakumar, M; Platt, FM	1
Moyses, C	1
Elstein, D; Zimran, A	1
Guerrera, M; Ladisch, S	1
Butters, TD; Dwek, RA; Platt, FM	1
Deckelbaum, RJ; Juliano, RA; Seo, T; Worgall, TS	1
Dwek, RA; Perry, VH; Platt, FM; Platt, N; Rudd, PM; Tian, G; Wilcockson, D	1
Aerts, JM; Butters, TD; Cox, TM; Dwek, RA; Elstein, D; Hollak, C; Hrebicek, M; Lachmann, RH; Maas, M; Platt, FM; van Weely, S; Zimran, A	1
Butters, TD; Dwek, RA; Platt, FM; Suganuma, R; van der Spoel, AC; Walden, CM; Yanagimachi, R	1
Antebi, V; Arnon, Y; Doniger, GM; Elstein, D; Guedalia, J; Simon, ES; Zimran, A	1
Garner, B; Gelissen, I; Glaros, EN; Jessup, W; Kim, WS; Quinn, CM; Wong, J	1
Cox, TM	1
Groot-Loonen, JJ; Hoogerbrugge, PM; Jacobs, JF; Wevers, RA; Willemsen, MA	1
Barnett, NL; Kolodny, EH; Pastores, GM	1
Barranger, JA; Charrow, J; Grabowski, GA; Mankin, HJ; Mistry, P; Weinreb, NJ	1
Butters, TD; Dwek, RA; Platt, FM; van der Spoel, AC; Walden, CM	1
Addobbati, R; Barone, R; Bembi, B; Butters, T; Cariati, R; Ciana, G; Fernandez-Guillen, L; Grasso, D; Guerci, VI; Marchetti, F; Pittis, MG	1
Lachmann, RH	1
Becq, F; Bijvelds, M; DeJonge, H; Jorna, H; Melin, P; Noel, S; Norez, C; Wilke, M	1
Dijkhuis, AJ; Kamps, W; Klappe, K; Kok, JW; Sietsma, H	1
Fujisawa, T; Hayakawa, T; Ishii, K; Ishitsuka, R; Ito, K; Kobayashi, T; Makino, A; Matsuo, H; Murate, M; Suzuki, M; Suzuki, Y	1
Acedo, A; Alfonso, P; Alonso, D; Barez, A; Corrales, A; Franco, R; Giraldo, P; Latre, P; Pocovi, M; Roldan, V; Serrano, S	1
Platt, FM; Svanborg, C; Svensson, M	1
Stirnemann, J	2
Germain, DP; Mistry, P	2
Aricescu, AR; Chang, VT; Crispin, M; Davis, SJ; Dwek, RA; Harvey, DJ; Jones, EY; Rudd, PM; Yu, C	1
Dhavale, DD; Karanjule, NS; Markad, SD; Sabharwal, SG; Sharma, T	1
Brady, RO	1
Beutler, E	1
Baker, R; Ginsberg, L; Goodwin, S; Hughes, DA; Mehta, AB; Milligan, A; Richfield, L	1
Amory, JK; Bhandari, A; Bone, W; Bremner, WJ; Leifke, E; Muller, CH; Page, ST; Pagel, ER; Radlmaier, A; Subramanyam, B	1
Alp, NJ; Butters, TD; Channon, KM; Clarke, K; Dwek, RA; Heare, T; Kulkarni, AB; Platt, FM; Priestman, DA; Qasba, P	1
Chung, S; Do, H; Kornfeld, S; Lee, WS; Pine, CW; Steet, R	1
Bone, W; Boomkamp, S; Fritsch, M; Gottwald, U; Leifke, E; Platt, FM; van der Spoel, AC; Voigtmann, U; Walden, CM	1
Goker-Alpan, O; Sidransky, E	1
Eto, Y	1
Alfonso, P; Capablo, JL; de Cabezón, AS; Franco, R; Giraldo, P; Pocovi, M	1
Collmann, A; Compostella, F; De Libero, G; Facciotti, F; Michieletti, M; Mori, L; Panza, L; Schümann, J	1
Hachulla, E; Javier, RM	2
Branza-Nichita, N; Durantel, D; Dwek, RA; Lazar, C; Macovei, A; Zitzmann, N; Zoulim, F	1
Antigny, F; Becq, F; Norez, C; Vandebrouck, C	1
Chen, SJ; Chien, YH; Huang, AC; Hwu, WL; Lee, NC; Peng, SF; Tsai, LK	1
Aerts, JF; Altarescu, G; Attias, D; Dweck, A; Elstein, D; Hadas-Halpern, I; van Weely, S; Zevin, S; Zimran, A	1
Clozel, M; Morand, O; Treiber, A	1
Aviezer, D; Brumshtein, B; Butters, TD; Futerman, AH; Greenblatt, HM; Shaaltiel, Y; Silman, I; Sussman, JL	1
Abel, L; Patterson, MC; Prady, H; Vecchio, D; Wraith, JE	1
Dingemanse, J; van Giersbergen, PL	1
Itoh, K; Tsuji, D	1
Elstein, D; Hrebícek, M; Pastores, GM; Zimran, A	1
Sedel, F	2
Jakóbkiewicz-Banecka, J; Wegrzyn, A; Wegrzyn, G	1
Butters, TD; Dwek, RA; Platt, FM; Priestman, DA; van der Spoel, AC	1
Pastores, GM	1
Froissart, R; Guffon, N; Maire, I	1
Belmatoug, N; de Villemeur, TB; Stirnemann, J	1
Mruk, DD	1
Becq, F; Bot, AG; De Jonge, HR; Noël, S; Wilke, M	1
Bembi, B; Deegan, P	1
Mehta, A	1
Jeyakumar, M; Platt, FM	1
Chippington, S; McHugh, K; Vellodi, A	1
Butters, TD; d'Azzo, A; Dwek, RA; Elliot-Smith, E; Jeyakumar, M; Lloyd-Evans, E; Platt, FM; Smith, DA; Speak, AO; van der Spoel, AC	1
Mott, R; Platt, FM; van der Spoel, AC	1
Baker, R; Berger, L; Holloway, B; Hughes, DA; Mehta, AB; Mikosch, P; Reed, M	1
Goodyear, CS; Greenshields, KN; Platt, FM; Speak, AO; Townson, KH; Willison, HJ	1
Burton, DR; Dwek, RA; Pollock, S; Zitzmann, N	1
Becq, F; Bezzerri, V; Borgatti, M; Cabrini, G; Dechecchi, MC; Gambari, R; Mancini, I; Nicolis, E; Norez, C; Ribeiro, CM; Rizzotti, P	1
Cordeiro, ML; Liberalesso, PB; Löhr, A; Raskin, S; Santos, ML; Telles, DS; Vieira, SC	1
Baumann, N; Billette de Villemeur, T; Feillet, F; Galanaud, D; Guffon, N; Heron, B; Lehéricy, S; Leveque, N; Sedel, F; Tourbah, A; Vanier, MT	1
Abel, L; Benko, W; Davies, EH; DeVile, C; Fitzgibbon, EJ; Harris, C; Ries, M; Schiffmann, R; Timmons, M; van Schaik, IN; Vellodi, A	1
Cohen, S; Coste, S; Guffon-Fouilhoux, N; Guillaumont, M; Guitton, J; Manchon, M	1
Antigny, F; Becq, F; Noel, S; Norez, C; Vandebrouck, C	1
Chérin, P; Giraldo, P; Mehta, A; Pastores, GM	1
Andria, G; Barone, MV; Cardone, M; Fontana, F; Parenti, G; Porto, C; Rossi, B; Tarallo, A; Tuzzi, MR	1
Leal, T; Lebacq, J; Lebecque, P; Leonard, A; Lubamba, B; Vanbever, R; Wallemacq, P	1
Gianutsos, J; Kolodny, EH; Luzy, C; Pastores, GM; Shapiro, BE	1
Banwell, B; Clarke, JT; Dingemanse, J; Maegawa, GH; Morgan, CP; Tifft, CJ; van Giersbergen, PL; Yang, S	1
Etou, Y	1
Bembi, B; Hollak, CE; Hughes, D; Schwierin, B; van Schaik, IN	1
Alfonso, P; Alonso, D; Atutxa, K; Barez, A; Fernández-Galán, MA; Franco, R; Giraldo, P; Latre, P; Martin, A; Pocovi, M	1
Bembi, B; Giorgino, R; Hwu, WL; Korenke, GC; Luzy, C; Marquardt, T; Mengel, E; Patterson, MC; Pineda, M; Rohrbach, M; Sedel, F; Walterfang, M; Wraith, JE	1
Ali, NF; Davidson, CD; Dobrenis, K; Micsenyi, MC; Ory, DS; Renault, S; Stephney, G; Vanier, MT; Walkley, SU	1
Aerts, JM; Belmatoug, N; Bembi, B; Cohen, Y; Collin-Histed, T; Cox, TM; Deegan, P; di Rocco, M; Giraldo, P; Hollak, CE; Hrebicek, M; Manuel, J; Mengel, E; Michelakakis, H; Parini, R; Pocovi, M; Reinke, J; Sa Miranda, MC; Tylki-Szymanska, A; van Dussen, L; vom Dahl, S; Zimran, A	1
Abel, L; Chadha-Boreham, H; Giorgino, R; Jacklin, E; Luzy, C; Patterson, MC; Vecchio, D; Wraith, JE	2
Dekomien, G; Lefeber, DJ; Morava, E; Wevers, RA; Willemsen, MA; Wortmann, SB	1
Berg, JE; Tallaksen, CM	1
Abegg, M; Barton, JJ; Lanyon, LJ; Mattman, A; Scheel, M	2
Alfonso, P; Gervas, J; Giraldo, P; Irun, P; Pocovi, M; Puzo, J	1
Coll, MJ; de la Barca, OB; Domingo, R; Fumero, LM; Gaspar, A; Macías-Vidal, J; O'Callaghan, M; Peñas, JJ; Pérez, AV; Perez-Poyato, MS; Pineda, M; Pocovi, M; Portal, LR; Roldán, S; Silva, MT; Temudo, T; Vilaseca, MA	1
Dvorakova, L; Gurda, B; Kothare, SV; Steinborn, B; Vlaskova, H; Zarowski, M	1
Marie, I	1
Amato, D; Piran, S	1
Becq, F; Billet, A; Callebaut, I; Jollivet, M; Melin, P; Mornon, JP	1
Vanier, MT	1
Chabrol, B	1
Bergmann, M; Gabius, HJ; Kopitz, J	1
Guitton, J; Masciullo, M; Modoni, A; Ricci, E; Santoro, M; Silvestri, G; Tonali, P	1
Champion, H; Cox, TM; Gallagher, J; Imrie, J; Lachmann, RH; Ramaswami, U; Wraith, JE	1
Lo, SM; McNamara, J; Mistry, PK; Seashore, MR	1
Fornfeist, S	2
Bianconi, S; Blennow, K; Fu, R; Månsson, JE; Mattsson, N; Porter, FD; Yanjanin, NM; Zetterberg, H	1
Hughes, DA; Pastores, GM	1
Amitrano, M; Andria, G; Astarita, L; Bruschini, D; Della Casa, R; Fecarotta, S; Parenti, G; Romano, A	1
Bin-Dorel, S; Decullier, E; Fouilhoux, A; Guffon, N; Guitton, J; Paillet, C	1
Berton, G; Bezzerri, V; Cabrini, G; Cioffi, F; Dechecchi, MC; Gambari, R; Huang, S; Lampronti, I; Mazzi, P; Nicolis, E; Scupoli, MT; Wiszniewski, L	1
Chen, CL; Chen, TY; Chen, YP; Cheng, YL; Hsieh, CY; Huang, WC; Lin, CF; Lin, CM; Lin, YS; Lu, PJ; Tsai, CC; Tsao, CW; Tseng, PC; Wang, CY; Wang, SH	1
Belmatoug, N; Burlina, A; Giraldo, P; Hendriksz, CJ; Kuter, DJ; Mengel, E; Pastores, GM	1
Czartoryska, B; Groener, JE; Jurkiewicz, E; Kamiński, ML; Tylki-Szymańska, A; Ługowska, A	1
Aoki, K; Eto, Y; Ida, H; Kaneshiro, E; Kawagoe, S; Kobayashi, H; Ohashi, T; Shimada, Y; Shimizu, H	1
Camou, F; Viallard, JF	1
Donald, A; Oko, R; van der Spoel, AC; Xu, W	1
Abian, O; Alfonso, P; Giraldo, P; Pocovi, M; Sancho, J; Velazquez-Campoy, A	1
Dingemanse, J; Spieker, E; Wagner-Redeker, W	1
Guthoff, R; Hovakimyan, M; Lukas, J; Maass, F; Petersen, J; Reichard, M; Rolfs, A; Stachs, O; Witt, M; Wree, A	1
Giraldo, P	1
Dahlman, I; Engvall, M; Hägglund, H; Hast, R; Klimkowska, M; Lerner, R; Machaczka, M	1
Hughes, D	1
Dingemanse, J; Leal, T; Lebecque, P; Leonard, A	1
Chien, YH; Huang, AC; Hwu, WL; Lee, NC; Peng, SF; Su, SC; Tsai, LK; Tseng, CC; Yang, CC	1
Bryan, C; Crooks, A; Ding, W; Dingemanse, J; O'Donnell, P; Prociuk, M; Sikora, T; Stein, VM; Vanier, MT; Vite, CH; Walkley, SU	1
McKiernan, P	1
Baruteau, J; Chabrol, B; Cuisset, JM; de Villemeur, TB; Dobbelaere, D; Eyer, D; Héron, B; Labarthe, F; Latour, P; Maurey, H; Ogier, H; Sedel, F; Valayannopoulos, V; Vanier, MT	1
Barone, R; Dardis, A; Di Rocco, M; Fiumara, A; Madeo, A	1
Klünemann, HH; Santosh, PJ; Sedel, F	1
Gordo, MM; Marfa, MP; Pérez-Poyato, MS	1
Biancini, GB; Coelho, JC; Mescka, CP; Negretto, G; Pires, R; Ribas, GS; Rodrigues, D; Vanzin, CS; Vargas, CR; Wajner, M; Wayhs, CA	1
Igdoura, SA; Venier, RE	1
Hibi, T; Miura, S; Mizushima, T; Nagata, H; Nakashita, M; Suzuki, H; Taki, T; Uehara, K	1
Amiri, M; Naim, HY	2
Chien, YH; Imrie, J; Patterson, MC; Rushton, D; Schubiger, D; Walterfang, M	1
Aryani, O; Ashrafi, MR; Ghofrani, M; Houshmand, M; Karimzadeh, P; Rahmanifar, A; Rezayi, A; Salehpour, S; Shafeghati, Y; Shiva, F; Shoar, BN; Taghdiri, MM; Tavasoli, A; Tonekaboni, SH; Zaman, T	1
Amato, D; Cox, TM; Giorgino, R; Hollak, CE; Luzy, C; Silkey, M; Steiner, RD	1
Drevon, H; Mengel, E; Muller, A; Patterson, MC; Pineda, M; Schwierin, B; Vanier, MT; Wijburg, FA	1
Ikenoya, S; Kitatani, T; Takahashi, S	1
Allman, SA; Alonzi, DS; Butters, TD; Dwek, RA; Hakki, Z; Kukushkin, NV; Pierce, L; Williams, SJ	1
Blank, PS; Cluzeau, CV; Cologna, SM; Dail, MK; Lieberman, AP; Porter, FD; Siebel, S; Toth, CL; Wassif, CA; Yanjanin, NM	1
Belmatoug, N; Brand, M; Giorgino, R; Giraldo, P; Hollak, CE; Hughes, D; Kuter, DJ; Mehta, A; Muller, A; Schaaf, B; Zimran, A	1
Alonzi, DS; Becq, F; Best, D; Blériot, Y; Butters, TD; Fleet, GW; Jenkinson, SF; Kato, A; Kunimatsu, T; Martínez, RF; Mui, J; Nakagawa, S; Norez, C; Saville, AW; Weymouth-Wilson, AC; Wilson, FX	1
Burton, DR; Caulfield, M; Clayton, T; Cupo, A; Deller, MC; Doores, KJ; Feizi, T; Hoffenberg, S; Hua, Y; Julien, JP; Khayat, R; King, CR; Klasse, PJ; Kong, L; Le, KM; Lee, JH; Liu, Y; Marozsan, A; McBride, R; Moore, JP; Murin, CD; Paulson, JC; Sanders, RW; Stanfield, RL; Tien, H; Ward, AB; Wilson, IA	1
Cai, YD; Chen, L; Huang, T; Jiang, M; Kong, XY; Li, BQ; Lu, J; Niu, B; Wei, ZJ; Zheng, MY	1
Bautista, J; Cáceres-Redondo, MT; Carrillo, F; Coll, MJ; Cordones, I; Escudero, M; Jesús, S; Macías-Vidal, J; Mir, P	1
Döme, P; Faludi, G; Gonda, X	1
Bagel, JH; Davidson, C; Fan, M; Fujiwara, H; Ory, DS; Porter, FD; Schaffer, JE; Sidhu, R; Tortelli, B; Vite, C; Walkley, SU; Yanjanin, NM; Zhang, J	1
Hadjivassiliou, M; Kheder, A; Olpin, S; Scott, C	1
Hanon, C; Maubert, A; Metton, JP	1
Frech, MJ; Holzmann, C; Hovakimyan, M; Hübner, R; Lukas, J; Maass, F; Petersen, J; Rolfs, A; Witt, M; Wree, A	1
Bembi, B; Bertini, E; Ceravolo, F; D'Amico, A; Dardis, A; Deodato, F; Dionisi-Vici, C; Ginocchio, VM; Verrigni, D	1
Andrejkovics, M; Balla, P; Balogh, I; Berényi, E; Lengyel, A; Magyar, A; Oláh, E; Szakszon, K; Szegedi, I	1
Lyseng-Williamson, KA	1
Bush, AI; Faux, NG; Firnkes, S; Ganio, G; Hastings, C; Hung, YH; Killilea, DW; Ory, DS; Porter, FD; Volitakis, I; Walterfang, M; Yanjanin, N	1
Edir, A; Guibbal, L; Hovnanian, A; Savignac, M; Simon, M	1
Butters, T; Deepak, S; Hall, CJ; Isenberg, DA; Jury, EC; Magee, AI; McDonald, G; Miguel, L	1
Al Eisa, N; Amraoui, Y; Arias, B; Beck, M; Cortina-Borja, M; Cousins, A; Cox, TM; Hartung, R; Hastings, C; Hendriksz, CJ; Imrie, J; Jacklin, E; Jeyakumar, M; Jiang, X; Lachmann, RH; Mengel, E; Ory, DS; Platt, FM; Porter, FD; Ramaswami, U; Riddick, K; Rimmele, F; Rolfs, A; Runz, H; Sidhu, R; Sillence, DJ; Simmons, L; Smith, DA; Speak, AO; Taylor, C; te Vruchte, D; Wallom, KL; Wassif, CA; Wraith, JE; Wright, N; Yanjanin, NM	1
Haÿ, E; Jacquot, JP; Le Henaff, C; Marie, PJ; Marty, C; Tabary, O; Velard, F	1
Hughes, DA; Mehta, A; Thomas, AS	1
Al Eisa, N; Platt, FM; Smith, C; Smith, DA; Wallom, KL; Williams, IM	1
Kaminsky, P; Lidove, O	1
Mauro, T	1
Becq, F; Boinot, C; Ferru-Clément, R; Jollivet Souchet, M	1
Agovino, T; Andria, G; Ascione, S; Danesino, C; De Filippi, P; Della Casa, R; Donati, MA; Fecarotta, S; la Marca, G; Mollica, C; Morandi, LO; Nusco, E; Ombrone, D; Parenti, G; Pasanisi, MB; Pichiecchio, A; Porto, C; Ravaglia, S; Romano, A; Rosa, M; Rossi, B; Sacchini, M	1
Aureli, M; Bassi, R; Bezzerri, V; Cabrini, G; Cantù, C; Cavazzini, A; Cheng, SH; Dechecchi, MC; Gambari, R; Giri, MG; Lampronti, I; Loberto, N; Lovato, V; Marchetti, N; Munari, S; Sonnino, S; Tebon, M	1
Gu, Y; Harvey, DJ; Hussain, S; McCauley, JW; Miller, JL; Rosenthal, PB; Zitzmann, N	1
Bembi, B; Calandra, S; Dardis, A; De Maglio, G; Di Muzio, A; Maruotti, V; Pizzolitto, S; Platt, F; Rabacchi, C; Sechi, A; Zampieri, S; Zanoni, P	1
Hawlitschka, A; Hovakimyan, M; Lukas, J; Maass, F; Petersen, J; Rolfs, A; Schmitt, O; Witt, M; Wree, A	1
Hopkin, RJ; Hufnagel, SB; Jefferies, JL; Karl, G; Pabón, LA; Prada, CE; Serrano, NC; Villamizar-Schiller, IT	1
Ambühl, M; Bandilla, D; Riahi, S; Stichler, J	1
Alsop, J; Bembi, B; Brand, M; Hughes, D; Muller, A; van Schaik, IN	1
Cardarelli, RA; Chen, Y; Gaval-Cruz, M; Higurashi, N; Hirose, S; Horiuchi, Y; Ishizuka, K; Kai, M; Kano, S; Kato, TA; Koga, M; Kondo, MA; Lee, Y; Maegawa, G; O'Donnell, P; Obie, C; Okano, H; Rapoport, JL; Sawa, A; Sedlak, TW; Seshadri, S; Srivastava, R; Tristan, C; Valle, D; Wilson, AM; Yuan, M	1
Bembi, B; Cox, TM; Deegan, P; Deroma, L; Hollak, C; Shemesh, E; Weinreb, NJ	1
Andreotti, G; Cubellis, MV; Monticelli, M	1
Alpa, M; Andria, G; Ardissone, A; Bembi, B; Bertini, E; Bruschini, D; Caviglia, S; D'Amico, A; Dardis, A; Del Giudice, E; Della Casa, R; Deodato, F; Di Rocco, M; Dionisi-Vici, C; Fecarotta, S; Federico, A; Filla, A; Fiumara, A; Gabrielli, O; Mansi, G; Palmeri, S; Parenti, G; Roccatello, D; Romano, A; Russo, C; Santoro, L; Uziel, G	1
Berezenko, VS; Dyba, MB	1
Alonzi, DS; Antonopoulos, A; Butters, TD; Chaidos, A; Danks, L; Dell, A; Dwek, RA; Ersek, A; Freidin, A; Goudevenou, K; Haslam, SM; Hatjiharissi, E; Horwood, NJ; Hu, M; Karadimitris, A; Papaioannou, M; Parry, S; Rahemtulla, A; Roberts, I; Santo, AE; Spanoudakis, E; Twigg, G; Vattakuzhi, Y; Williams, LM; Xu, K	1
Cornelisse, P; Mengel, E; Muller, A; Patterson, MC; Pineda, M; Schwierin, B; Vanier, MT	1
Amato, D; Chadha-Boreham, H; Marquardt, T; Morand, O; Remenova, T; Tsurutani, S	1
Abel, L; Bowman, EA; Desmond, P; Fahey, M; Velakoulis, D; Walterfang, M	1
Serratrice, C; Serratrice, J; Swiader, L	1
Alvarez-Fischer, D; Andreas, H; Hirsch, EC; Höglinger, GU; Höllerhage, M; Lu, L; Noelker, C; Oertel, WH; Roscher, R; Sturn, A; Vulinovic, F	1
Barone, R; Di Rocco, M; Fiumara, A; Madeo, A	1
Amaral, MM; Girard, MC; Ibarra, C; Repetto, HA; Rivera, FP; Sacerdoti, F	1
Claridge, TD; Edgar, M; Grootveld, M; Lachmann, RH; Platt, FM; Probert, F; Ruiz-Rodado, V; te Vruchte, D; Tocchio, AZ; Zhang, X	1
Abel, LA; Bowman, EA; Stainer, MJ; Velakoulis, D; Walterfang, M	1
Cardinale, A; Cutarelli, A; D'Arcangelo, G; Frank, C; Grossi, D; Merlo, D; Racaniello, M; Rufini, S; Tancredi, V; Zaratti, A	1
Berman, JN; Forrester, AM; Sultana, S; Truong, NY; van der Spoel, AC; Veinotte, CJ; Vieira, DB; Wigger, JG	1
Audoin, B; Burzykowski, T; Chabrol, B; Galanaud, D; Kaphan, E; Sedel, F; Tourbah, A; Tranchant, C; Vanier, MT	1
Aerts, JM; Ferraz, MJ; Hollak, CE; Mirzaian, M; Overkleeft, HS; Smid, BE; Verhoek, M; Wisse, P	1
Byers, S; Derrick-Roberts, A; Fletcher, J; Kaidonis, X; Ranieri, E; Sharp, P	1
Balter, R; Banzato, C; Bordugo, A; Dardis, A; Degani, D; Giordano, G; Gugelmo, G; Maines, E; Tadiotto, E	1
Burin, M; de Mari, J; Deon, M; Giugliani, R; Kessler, R; Mescka, C; Michelin, K; Ribas, GS; Saraiva-Pereira, ML; Souza, HM; Trapp, F; Vargas, CR	1
Degtyareva, AV; Mikhailova, SV; Puchkova, AA; Tumanova, EL; Zakharova, EY	1
Barrier, L; Ingrand, S; Noel, A	1
Alves, S; Coutinho, MF; Santos, JI	1
Cuisset, JM; Dobbelaere, D; Latour, P; Michaud, L; Sukno, S; Trauffler, A; Vallée, L	1
Di Francesco, E; Di Raimondo, F; Fiumara, A; Giuffrida, G; Lombardo, R; Parrinello, L	1
Grittner, U; Holzmann, C; Rolfs, A; Schlegel, V; Thieme, M; Witt, M; Wree, A	1
Acedo, A; Alfonso, P; Andrade-Campos, M; Atutxa, K; Barez, A; Blanes, M; de la Serna, J; Diaz-Morant, V; Fernández-Galán, MA; Franco, R; Gil-Cortes, C; Giner, V; Giraldo, P; Hernández-Martin, R; Ibañez, A; Irun, P; Latre, P; Loyola, I; Luño, E; Medrano-Engay, B; Pocovi, M; Puerta, J; Roig, I; Salamero, O; Villalón, L	1
Agans, KN; Callahan, MV; Garza, NL; Geisbert, JB; Geisbert, TW; Klose, B; Mire, CE; Qiu, X; Ramstedt, U; Shurtleff, AC; Stuthman, KS; Treston, AM; Van Tongeren, SA; Warfield, KL; Warren, TK; Wells, J; Wong, G	1
Ceravolo, F; Concolino, D; Falvo, F; Grisolia, M; Moricca, MT; Sestito, S	1
Canda, E; Coker, M; Kagnici, M; Kose, M; Sozmen, EY; Ucar, SK	1
Adanyeguh, I; Galanaud, D; Masingue, M; Mochel, F; Nadjar, Y; Sedel, F	1
Ganz, ML; Hamed, A; Nalysnyk, L; Selzer, M; Stern, S; Ward, A; Weinreb, N	1
Günther, R; Holzmann, C; Meyer, A; Rolfs, A; Schmitt, O; Witt, M; Wree, A	1
Jarnes Utz, JR; Kim, S; King, K; Redtree, ES; Schema, L; Whitley, CB; Ziegler, R	1
Stellitano, LA; Verity, CM; Winstone, AM	1
Caciotti, A; Deodato, F; Dionisi-Vici, C; Donati, MA; Morrone, A; Procopio, E; Rampazzo, A; Scarpa, M; Taurisano, R	1
Adachi, K; Itamura, S; Jimbo, E; Miyauchi, A; Nakamura, S; Nakano, Y; Nanba, E; Narita, A; Osaka, H; Usui, M; Yamagata, T	1
Boudewyn, LC; Dobrenis, K; Grishchuk, Y; Kuchar, L; Ledvinova, J; Sikora, J; Walkley, SU; Wang, SL	1
Claridge, TDW; Farhat, N; Grootveld, M; Nicoli, ER; Platt, FM; Porter, FD; Probert, F; Ruiz-Rodado, V; Vruchte, DT; Wassif, CA	1
Abe, K; Sakai, N	1
Elben, S; Groiss, SJ; Hassan, SS; Schnitzler, A; Trenado, C	1
Cobucci-Ponzano, B; D'Alonzo, D; De Fenza, M; Guaragna, A; Huebecker, M; Iacono, R; Moracci, M; Palumbo, G; Parenti, G; Platt, F; Porto, C; Priestman, DA	1
Bonatto, MP; Burin, MG; de Oliveira Schmitt Ribas, G; Giugliani, R; Hammerschmidt, TG; Kessler, RG; Michelin-Tirelli, K; Saraiva-Pereira, ML; Souza, FTS; Trapp, F; Vargas, CR	1
Bandaru, VVR; Dawson, TM; Dawson, VL; Karuppagounder, SS; Kim, D; Kim, S; Ko, HS; Kwon, SH; Lee, G; Lee, H; Lee, S; Mao, X; Pandey, A; Rhee, P; Umanah, GE; Yin, X; Yun, SP	1
Amato, D; Patterson, MA	1
De Rosa, A; Dubbioso, R; Esposito, M; Iodice, R; Manganelli, F; Ruggiero, L; Saccà, F; Santoro, L; Spina, E; Topa, A; Tozza, S	1
Patterson, MC; Pineda, M; Walterfang, M	1
Anheim, M; Audoin, B; Ayrignac, X; Cintas, P; De Gaillarbois, TD; Degardin, A; Deramecourt, V; Goizet, C; Héron, B; Hütter-Moncada, AL; Kaphan, E; Lamari, F; Latour, P; Laurencin, C; Martzolff, L; Nadjar, Y; Roze, E; Tilikete, C; Tranchant, C; Vanier, MT	1
Eratne, D; Farrand, S; Goh, AMY; Kelso, W; Mangelsdorf, S; Rego, T; Velakoulis, D; Walterfang, M	1
Insua, JL; Juríčková, K; Karimzadeh, P; Kolb, SA; Kolnikova, M; Malinova, V; Pineda, M; Velten, C	1
Do, HV; Feng, J; Frascella, M; Garcia, A; Gotschall, R; Khanna, R; Lun, Y; Martina, JA; Nair, A; Ponery, AS; Puertollano, R; Raben, N; Ralston, E; Schilling, A; Soska, R; Tuske, S; Valenzano, KJ; Valle, MCD; Xu, S	1
Anderson, K; Griffiths, TD; Lad, M; Thomas, RH	1
Bräuer, AU; Burstein, C; Engelmann, R; Frank, M; Gläser, A; Gräler, M; Müller-Hilke, B; Neidhardt, J; Neßlauer, AM; Rolfs, A; Witt, M; Wree, A	1
Adriaenssens, E; Bembi, B; Colaco, A; Dardis, A; Davis, LC; Deegan, PB; Fernández-Suárez, ME; Galione, A; Kaya, E; Platt, FM; Porter, FD; Tan, CY; Zampieri, S	1
Ardissone, A; Caciotti, A; Calamai, M; Casini, A; Deodato, F; Di Rocco, M; Fischetto, R; Fiumara, A; Guerrini, R; Mancardi, MM; Mangone, G; Marangi, A; Morrone, A; Parini, R; Pavone, FS; Procopio, E; Ricci, S; Salviati, A; Strisciuglio, P; Tonin, R	1
Antel, JP; Avila-Pacheco, J; Blain, M; Borucki, DM; Chao, CC; Clish, CB; de Lima, KA; Gutiérrez-Vázquez, C; Hewson, P; Lipof, GZ; Liu, L; Mayo, L; Prat, A; Quintana, FJ; Rothhammer, V; Sanmarco, LM; Takenaka, MC; Tjon, EC; Trauger, SA; Wheeler, MA; Zandee, SEJ	1
Bi, FC; Chen, DK; Dai, GY; Li, KE; Liu, Z; Rong, C; Yao, N; Yin, J	1
Arima, H; Fukaura, M; Hayashi, A; Higaki, K; Higashi, T; Hoque, S; Irie, T; Ishitsuka, Y; Komiya, T; Kondo, Y; Motoyama, K; Sakata, N; Yamada, Y	1
Brogden, G; Das, AM; Maalouf, K; Naim, HY; Rizk, S; Shammas, H; Walters, F	1
Garver, WS; Giugliani, R; Héron, B; Imrie, J; Jahnova, H; Meaney, FJ; Moneuse, P; Morand, O; Nadjar, Y; Patterson, MC; Rosenberg, D; Schwierin, B; Vanier, MT	1
Mengel, E; Moneuse, P; Patterson, MC; Pineda, M; Rosenberg, D; Vanier, MT	1
Barrio, SC; Extremera, VC; Gutiérrez-Solana, LG; Jiménez, IB; la Concepción Fournier Del Castillo, M; Marín, LL; Orduña, BE	1
D'Alonzo, D; Dechecchi, MC; Esposito, A; Fenza, M; Gregorio, E; Guaragna, A; Lippi, G; Tamanini, A	1
Calì, F; Costanzo, MC; Fiumara, A; Musumeci, SA; Nicotera, AG; Vinci, M; Vitello, A	1
Keage, M; Lewis, C; Schubiger, D; Velakoulis, D; Vogel, AP; Walterfang, M; Watanabe, M	1
Di Rocco, M; Favia, V; Fischetto, R; Gaeta, A; Giacomini, T; Giordano, P; Lassandro, G; Mancardi, MM; Morrone, A; Palladino, S; Palladino, V; Romano, A; Severino, M; Strisciuglio, P; Tripaldi, ME; Zampini, L	1
Alméciga-Díaz, CJ; Benincore-Flórez, E; Echeverri-Peña, OY; Espejo-Mojica, AJ; Garzón Jaramillo, RG; Leal, AF; Solano-Galarza, D; Suarez, DA	1
Farhat, N; King, MC; Machielse, L; Porter, FD; Sinaii, N; Smith, AC; Solomon, BI	1
Eto, K; Hamada, Y; Han, G; Imanishi, A; Kanbayashi, T; Kawazoe, T; Kondo, H; Kumagai, T; Mishima, K; Noguchi, A; Sakai, N; Shimizu, T; Takahashi, T; Tsutsui, K	1
Antipova, V; Holzmann, C; Rolfs, A; Witt, M; Wree, A	1
Çakar, NE; Önal, H	1
Kazungu, Y; Marcello, A; Martinez-Orellana, P; Milan Bonotto, R; Nascimento Alves, L; Poggianella, M; Polez, S; Rajasekharan, S; Skoko, N	1
Bratkovic, D; Byrne, BJ; Castelli, J; Díaz-Manera, J; Goldman, M; Jiang, H; Kishnani, PS; Kuchipudi, S; Laforêt, P; Mozaffar, T; Roberts, M; Schoser, B; Sitaraman, S; Toscano, A; van der Ploeg, AT	1
Donida, B; Dos Reis, BG; Faverzani, JL; Hammerschmidt, TG; Kessler, RG; Machado, AZ; Moura, AP; Moura, DJ; Reinhardt, LS; Sebastião, FM; Vargas, CR	1
Böger, B; Fachi, MM; Leonart, LP; Lombardi, NF; Pedroso, MLA; Pontarolo, R; Silva, MRD; Szpak, R	1
Gutić, M; Janković, SM; Milosavljević, MN	1
Akbostancı, MC; Ceylaner, S; Elibol, B; Erdoğan, S; Yalçın Çakmaklı, G	1
Blair, HA	1
Ashrafi, MR; Bemanalizadeh, M; Dakkali, MS; Daneshfar, S; Heidari, M; Hosseinpour, S; Khodarahmi, M; Mansouri, V; Sharifianjazi, F; Tavasoli, AR	1
Bennett, JJ; Murphy, PV	1
Brassier, A; Broué, P; Cances, C; Chabrol, B; Dahmani-Rabehi, B; Eyer, D; Freihuber, C; Héron, B; Labarthe, F; Latour, P; Levade, T; Pichard, S; Sevin, C; Vanier, MT	1

Reviews

56 review(s) available for 1-deoxynojirimycin and miglustat

Article	Year
The many faces of the adamantyl group in drug design. European journal of medicinal chemistry, 2011, Volume: 46, Issue:6 Topics: Adamantane; Animals; Antimalarials; Diabetes Mellitus, Type 2; Drug Design; Humans; Hypoglycemic Agents; Iron Chelating Agents; Iron Overload; Malaria; Solubility	2011
Pharmacological chaperones as therapeutics for lysosomal storage diseases. Journal of medicinal chemistry, 2013, Apr-11, Volume: 56, Issue:7 Topics: Cell Line; Glycolipids; Humans; Lysosomal Storage Diseases; Molecular Chaperones; Structure-Activity Relationship	2013
Biology and potential strategies for the treatment of GM2 gangliosidoses. Molecular medicine today, 1998, Volume: 4, Issue:4 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Animals; beta-N-Acetylhexosaminidases; Bone Marrow Transplantation; Cats; Cell Transplantation; Child; Disease Models, Animal; Dogs; G(M2) Ganglioside; Genetic Therapy; Genetic Vectors; Glycolipids; HIV; Humans; Infant; Lysosomes; Mice; Mice, Knockout; Neurons; Phenotype; Point Mutation; Rats; Sandhoff Disease; Swine; Tay-Sachs Disease; Transplantation, Homologous	1998
The molecular basis of lysosomal storage diseases and their treatment. Biochemical Society transactions, 2000, Volume: 28, Issue:2 Topics: 1-Deoxynojirimycin; Animals; Endocytosis; Enzyme Inhibitors; Genetic Therapy; Genotype; Humans; Lysosomal Storage Diseases; Lysosomes; Phenotype	2000
Stemming the tide: glycosphingolipid synthesis inhibitors as therapy for storage diseases. Glycobiology, 2000, Volume: 10, Issue:12 Topics: 1-Deoxynojirimycin; Clinical Trials as Topic; Glycosphingolipids; Humans; Lysosomal Storage Diseases; Propanolamines; Pyrrolidines	2000
Substrate reduction therapy for glycosphingolipid storage disorders. Expert opinion on investigational drugs, 2001, Volume: 10, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Glycosphingolipids; Humans; Lysosomal Storage Diseases; Mice	2001
Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis. Neuropathology and applied neurobiology, 2002, Volume: 28, Issue:5 Topics: 1-Deoxynojirimycin; Animals; Bone Marrow Transplantation; Chemotherapy, Adjuvant; Disease Models, Animal; G(M2) Ganglioside; Gangliosides; Glucosylceramides; Glucosyltransferases; Glycosphingolipids; Humans; Lysosomal Storage Diseases; Lysosomes; Mice; Models, Biological; Models, Chemical; Morpholines; Sandhoff Disease; Tay-Sachs Disease; Treatment Outcome	2002
Substrate reduction therapy in mouse models of the glycosphingolipidoses. Philosophical transactions of the Royal Society of London. Series B, Biological sciences, 2003, May-29, Volume: 358, Issue:1433 Topics: 1-Deoxynojirimycin; Animals; Disease Models, Animal; Enzyme Inhibitors; Mice; Sphingolipidoses; Substrate Specificity	2003
Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease. Philosophical transactions of the Royal Society of London. Series B, Biological sciences, 2003, May-29, Volume: 358, Issue:1433 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Glucosyltransferases; Humans; Substrate Specificity	2003
Gaucher disease and the clinical experience with substrate reduction therapy. Philosophical transactions of the Royal Society of London. Series B, Biological sciences, 2003, May-29, Volume: 358, Issue:1433 Topics: 1-Deoxynojirimycin; Clinical Trials as Topic; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Humans; Substrate Specificity	2003
New therapeutics for the treatment of glycosphingolipid lysosomal storage diseases. Advances in experimental medicine and biology, 2003, Volume: 535 Topics: 1-Deoxynojirimycin; Animals; Disease Models, Animal; Drug Therapy, Combination; Enzyme Inhibitors; Gaucher Disease; Glycosphingolipids; Humans; In Vitro Techniques; Lysosomal Storage Diseases; Mice	2003
Substrate reduction therapy for lysosomal storage diseases. Acta paediatrica (Oslo, Norway : 1992). Supplement, 2005, Volume: 94, Issue:447 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Glucosylceramidase; Glucosylceramides; Glycoside Hydrolases; Glycosphingolipids; Humans; Lysosomal Storage Diseases; Substrate Specificity	2005
Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. American journal of hematology, 2005, Volume: 80, Issue:3 Topics: 1-Deoxynojirimycin; Contraindications; Gaucher Disease; Glucosylceramidase; Glucosyltransferases; Humans; Practice Guidelines as Topic	2005
Miglustat: substrate reduction therapy for glycosphingolipid lysosomal storage disorders. Drugs of today (Barcelona, Spain : 1998), 2006, Volume: 42, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Clinical Trials as Topic; Drug Evaluation, Preclinical; Enzyme Inhibitors; Gaucher Disease; Glucosyltransferases; Glycosphingolipids; Humans; Lysosomal Storage Diseases	2006
Glycolipid receptor depletion as an approach to specific antimicrobial therapy. FEMS microbiology letters, 2006, Volume: 258, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Bacterial Adhesion; Escherichia coli Infections; Glycosphingolipids; Humans; Immunity, Innate; Lysosomal Storage Diseases; Receptors, Cell Surface; Urinary Tract Infections	2006
Emerging strategies for the treatment of hereditary metabolic storage disorders. Rejuvenation research, 2006,Summer, Volume: 9, Issue:2 Topics: 1-Deoxynojirimycin; Bone Marrow Transplantation; Enzyme Inhibitors; Fabry Disease; Gangliosidosis, GM1; Gaucher Disease; Genetic Therapy; Humans; Imino Sugars; Piperidines	2006
Gaucher disease: multiple lessons from a single gene disorder. Acta paediatrica (Oslo, Norway : 1992). Supplement, 2006, Volume: 95, Issue:451 Topics: 1-Deoxynojirimycin; Gaucher Disease; Globosides; Humans; Liver; Molecular Chaperones; Splenectomy	2006
[Is it possible to treat the brain? Recent advances in inherited neurodegenerative disorders]. No to hattatsu = Brain and development, 2007, Volume: 39, Issue:2 Topics: 1-Deoxynojirimycin; Bone Marrow Transplantation; Cell- and Tissue-Based Therapy; Enzyme Inhibitors; Enzyme Therapy; Genetic Therapy; Glucosamine; Heredodegenerative Disorders, Nervous System; Humans; Stem Cell Transplantation	2007
[Osteoarticular manifestations of Gaucher disease in adults: pathophysiology and treatment]. Presse medicale (Paris, France : 1983), 2007, Volume: 36, Issue:12 Pt 3 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Aged, 80 and over; Bone Density; Bone Diseases; Bone Diseases, Metabolic; Child; Child, Preschool; Clinical Trials as Topic; Diphosphonates; Enzyme Inhibitors; Fractures, Bone; Gaucher Disease; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Humans; Joint Diseases; Kyphosis; Magnetic Resonance Imaging; Middle Aged; Osteoarthritis; Osteonecrosis; Osteoporosis; Radionuclide Imaging; Spinal Fractures; Tomography, X-Ray Computed	2007
[Molecular pathogenesis and therapeutic targets of lysosomal diseases]. Seikagaku. The Journal of Japanese Biochemical Society, 2007, Volume: 79, Issue:7 Topics: 1-Deoxynojirimycin; Animals; Anti-Inflammatory Agents, Non-Steroidal; Aspirin; beta-N-Acetylhexosaminidases; Bone Marrow Transplantation; Disease Models, Animal; G(M2) Activator Protein; Genetic Therapy; Humans; Lysosomal Storage Diseases, Nervous System; Mutation	2007
Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies. Clinical therapeutics, 2007, Volume: 29, Issue:8 Topics: 1-Deoxynojirimycin; Adult; Bone Density; Bone Diseases, Metabolic; Enzyme Inhibitors; Female; Gaucher Disease; Glucosyltransferases; Humans; Male; Middle Aged; Multicenter Studies as Topic; Pain; Pain Measurement; Time Factors; Treatment Outcome	2007
Substrate deprivation therapy: a new hope for patients suffering from neuronopathic forms of inherited lysosomal storage diseases. Journal of applied genetics, 2007, Volume: 48, Issue:4 Topics: 1-Deoxynojirimycin; Animals; Enzyme Inhibitors; Gaucher Disease; Genistein; Humans; Lysosomal Storage Diseases; Lysosomes; Mucopolysaccharidosis III; Niemann-Pick Diseases; Sandhoff Disease; Tay-Sachs Disease	2007
Miglustat: substrate reduction therapy for lysosomal storage disorders associated with primary central nervous system involvement. Recent patents on CNS drug discovery, 2006, Volume: 1, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Brain; Brain Diseases; Enzyme Inhibitors; Humans; Lysosomal Storage Diseases	2006
[Therapeutic objectives in Gaucher disease]. La Revue de medecine interne, 2007, Volume: 28 Suppl 2 Topics: 1-Deoxynojirimycin; Adult; Child; Clinical Trials as Topic; Enzyme Inhibitors; Evidence-Based Medicine; Female; Gaucher Disease; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Humans; Male; Phenotype; Quality of Life; Recombinant Proteins; Time Factors; Treatment Outcome	2007
[Osteoarticular manifestations of Gaucher disease in adults: pathophysiology and treatment]. La Revue de medecine interne, 2007, Volume: 28 Suppl 2 Topics: 1-Deoxynojirimycin; Adult; Bone Density; Bone Diseases; Diphosphonates; Enzyme Inhibitors; Gaucher Disease; Genotype; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Humans; Joint Diseases; Magnetic Resonance Imaging; Osteonecrosis; Quality of Life; Recombinant Proteins; Registries; Time Factors; Treatment Outcome	2007
[Current development and usefulness of biomarkers for Gaucher disease follow up]. La Revue de medecine interne, 2007, Volume: 28 Suppl 2 Topics: 1-Deoxynojirimycin; Adult; Biomarkers; Chemokines, CC; Disease Progression; Enzyme Inhibitors; Female; Follow-Up Studies; Gaucher Disease; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Hexosaminidases; Humans; Immunohistochemistry; Infant, Newborn; Peptidyl-Dipeptidase A; Pregnancy; Pregnancy Complications; Recombinant Proteins; Time Factors	2007
New perspectives in non-hormonal male contraception. Trends in endocrinology and metabolism: TEM, 2008, Volume: 19, Issue:2 Topics: 1-Deoxynojirimycin; Animals; Calcium Channel Blockers; Contraception; Contraception, Immunologic; Contraceptive Agents, Male; Dimethyl Sulfoxide; Drug Design; Humans; Hydrazines; Indazoles; Indenes; Male; Maleates; Models, Biological; Nifedipine; Polyesters; Polystyrenes; Pyridines; Styrenes	2008
Gaucher disease: unmet treatment needs. Acta paediatrica (Oslo, Norway : 1992), 2008, Volume: 97, Issue:457 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Health Services Accessibility; Hematologic Neoplasms; Humans; Knee Joint; Magnetic Resonance Imaging; Recombinant Proteins	2008
Substrate reduction therapy. Acta paediatrica (Oslo, Norway : 1992), 2008, Volume: 97, Issue:457 Topics: 1-Deoxynojirimycin; Animals; Blood-Brain Barrier; Central Nervous System Diseases; Enzyme Inhibitors; Gaucher Disease; Glycoside Hydrolase Inhibitors; Humans; Lysosomal Storage Diseases; Niemann-Pick Disease, Type C; Treatment Outcome	2008
Differential sensitivity of mouse strains to an N-alkylated imino sugar: glycosphingolipid metabolism and acrosome formation. Pharmacogenomics, 2008, Volume: 9, Issue:6 Topics: 1-Deoxynojirimycin; Acrosome; Animals; Glycoside Hydrolase Inhibitors; Glycosphingolipids; Male; Mice; Mice, Inbred Strains; Models, Molecular; Molecular Structure; Pharmacogenetics; Species Specificity; Spermatogenesis; Spermatozoa	2008
Goal-oriented therapy with miglustat in Gaucher disease. Current medical research and opinion, 2009, Volume: 25, Issue:1 Topics: 1-Deoxynojirimycin; Adult; Aged; Bone and Bones; Female; Gaucher Disease; Hepatomegaly; Humans; Lung; Male; Middle Aged; Splenomegaly	2009
[Recent therapeutics for Fabry disease]. Nihon Naika Gakkai zasshi. The Journal of the Japanese Society of Internal Medicine, 2009, Apr-10, Volume: 98, Issue:4 Topics: 1-Deoxynojirimycin; alpha-Galactosidase; Cell- and Tissue-Based Therapy; Fabry Disease; Genetic Therapy; Glucosamine; Humans; Isoenzymes; Mass Screening; Trihexosylceramides	2009
Gaucher disease: a systematic review and meta-analysis of bone complications and their response to treatment. Journal of inherited metabolic disease, 2010, Volume: 33, Issue:3 Topics: 1-Deoxynojirimycin; Adult; Bone and Bones; Bone Density; Bone Marrow; Enzyme Replacement Therapy; Gaucher Disease; Humans; Lumbar Vertebrae; Magnetic Resonance Imaging; Time Factors; Treatment Outcome	2010
Niemann-Pick disease type C. Orphanet journal of rare diseases, 2010, Jun-03, Volume: 5 Topics: 1-Deoxynojirimycin; Age Factors; Age of Onset; Biological Transport; Cholesterol; Genetic Association Studies; Genetic Testing; Humans; Niemann-Pick Disease, Type C	2010
[Clinical diagnosis of the adult form of Niemann-Pick type C disease]. Archives de pediatrie : organe officiel de la Societe francaise de pediatrie, 2010, Volume: 17 Suppl 2 Topics: 1-Deoxynojirimycin; Adult; Cerebellar Ataxia; Cognition Disorders; Deglutition Disorders; Dysarthria; Enzyme Inhibitors; Epilepsy; Humans; Mental Disorders; Movement Disorders; Niemann-Pick Disease, Type C; Splenomegaly; Supranuclear Palsy, Progressive	2010
[Therapies in the Niemann-Pick type C disease]. Archives de pediatrie : organe officiel de la Societe francaise de pediatrie, 2010, Volume: 17 Suppl 2 Topics: 1-Deoxynojirimycin; Antidepressive Agents; Cataplexy; Diet; Enzyme Inhibitors; Humans; Niemann-Pick Disease, Type C	2010
The pathophysiology of GD - current understanding and rationale for existing and emerging therapeutic approaches. Wiener medizinische Wochenschrift (1946), 2010, Volume: 160, Issue:23-24 Topics: 1-Deoxynojirimycin; Animals; Autophagy; Calcium; Cell Membrane; Disease Models, Animal; DNA Mutational Analysis; Endoplasmic Reticulum; Enzyme Inhibitors; Enzyme Replacement Therapy; Gaucher Disease; Genetic Therapy; Glucosylceramidase; Glucosylceramides; Glycolipids; Homeostasis; Humans; Mice; Oxidative Stress; Proteostasis Deficiencies	2010
Gastrointestinal disturbances and their management in miglustat-treated patients. Journal of inherited metabolic disease, 2011, Volume: 34, Issue:5 Topics: 1-Deoxynojirimycin; Adult; Child; Clinical Trials as Topic; Drug-Related Side Effects and Adverse Reactions; Enzyme Inhibitors; Gastrointestinal Diseases; Gaucher Disease; Humans; Models, Biological	2011
Treatable metabolic psychoses that go undetected: what Niemann-Pick type C can teach us. International journal of psychiatry in clinical practice, 2012, Volume: 16, Issue:3 Topics: 1-Deoxynojirimycin; Age of Onset; Ataxia; Diagnosis, Differential; Disease Progression; Enzyme Inhibitors; Genetic Testing; Humans; Magnetic Resonance Spectroscopy; Niemann-Pick Disease, Type C; Ophthalmoplegia; Prevalence; Psychotic Disorders; Splenomegaly; Treatment Outcome	2012
Miglustat as a therapeutic agent: prospects and caveats. Journal of medical genetics, 2012, Volume: 49, Issue:9 Topics: 1-Deoxynojirimycin; Animals; Clinical Trials as Topic; Disease Models, Animal; Gangliosides; Gaucher Disease; Humans	2012
Dysphagia as a risk factor for mortality in Niemann-Pick disease type C: systematic literature review and evidence from studies with miglustat. Orphanet journal of rare diseases, 2012, Oct-06, Volume: 7 Topics: 1-Deoxynojirimycin; Animals; Deglutition Disorders; Humans; Niemann-Pick Disease, Type C	2012
[Pharmacological and clinical profiles of miglustat (Brazaves(®)) for the treatment of Niemann-Pick type C disease]. Nihon yakurigaku zasshi. Folia pharmacologica Japonica, 2013, Volume: 141, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Calcium; Clinical Trials as Topic; Disease Models, Animal; Enzyme Inhibitors; Glucosyltransferases; Humans; Lysosomes; Mice; Niemann-Pick Disease, Type C	2013
[Adult psychiatric aspects of Niemann-Pick disease]. Neuropsychopharmacologia Hungarica : a Magyar Pszichofarmakologiai Egyesulet lapja = official journal of the Hungarian Association of Psychopharmacology, 2013, Volume: 15, Issue:2 Topics: 1-Deoxynojirimycin; Age of Onset; Cataplexy; Cognition Disorders; Enzyme Inhibitors; Humans; Niemann-Pick Disease, Type A; Niemann-Pick Disease, Type B; Niemann-Pick Disease, Type C; Niemann-Pick Diseases; Sphingolipids; Sphingomyelin Phosphodiesterase	2013
[Adult onset Niemann-Pick type C disease and psychosis: literature review]. L'Encephale, 2013, Volume: 39, Issue:5 Topics: 1-Deoxynojirimycin; Adult; Carrier Proteins; Cooperative Behavior; Dementia; Early Diagnosis; Enzyme Inhibitors; Follow-Up Studies; France; Glycoproteins; Humans; Interdisciplinary Communication; Intracellular Signaling Peptides and Proteins; Membrane Glycoproteins; Neurologic Examination; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Psychiatry; Psychotic Disorders; Schizophrenia; Schizophrenic Psychology; Sequence Analysis, DNA; Vesicular Transport Proteins	2013
Miglustat: a review of its use in Niemann-Pick disease type C. Drugs, 2014, Volume: 74, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Enzyme Inhibitors; Humans; Niemann-Pick Disease, Type C	2014
Gaucher disease: haematological presentations and complications. British journal of haematology, 2014, Volume: 165, Issue:4 Topics: 1-Deoxynojirimycin; Anemia; Combined Modality Therapy; Disease Management; Enzyme Replacement Therapy; Gaucher Disease; Genetic Predisposition to Disease; Glucosylceramidase; Glycosphingolipids; Hemorrhagic Disorders; Humans; Inflammation; Lewy Body Disease; Lysosomes; Macrophage Activation; Multiple Myeloma; Parkinson Disease; Splenectomy; Splenomegaly; Thrombocytopenia; Unfolded Protein Response	2014
[Current therapeutic strategies in lysosomal disorders]. Presse medicale (Paris, France : 1983), 2014, Volume: 43, Issue:11 Topics: 1-Deoxynojirimycin; Codon, Nonsense; Codon, Terminator; Cystine Depleting Agents; Enzyme Inhibitors; Enzyme Replacement Therapy; Gene Expression Regulation; Hematopoietic Stem Cell Transplantation; Humans; Imino Pyranoses; Lysosomal Storage Diseases; Mutant Proteins; Renal Insufficiency	2014
Enzyme replacement and substrate reduction therapy for Gaucher disease. The Cochrane database of systematic reviews, 2015, Mar-27, Issue:3 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Enzyme Replacement Therapy; Gaucher Disease; Glucosylceramidase; Hemoglobin A; Hepatomegaly; Humans; Platelet Count; Randomized Controlled Trials as Topic; Splenomegaly; Substrate Specificity	2015
Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders. International journal of molecular sciences, 2016, Jul-04, Volume: 17, Issue:7 Topics: 1-Deoxynojirimycin; Enzyme Replacement Therapy; Gaucher Disease; Genistein; Humans; Lysosomal Storage Diseases; Mucopolysaccharidoses; Niemann-Pick Disease, Type C	2016
Miglustat in Niemann-Pick disease type C patients: a review. Orphanet journal of rare diseases, 2018, 08-15, Volume: 13, Issue:1 Topics: 1-Deoxynojirimycin; Biomarkers; Enzyme Inhibitors; Humans; Niemann-Pick Disease, Type C; Treatment Outcome	2018
Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management. CNS drugs, 2019, Volume: 33, Issue:2 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; Brain; Cognitive Dysfunction; Diagnosis, Differential; GABAergic Neurons; Humans; Models, Neurological; Neuronal Plasticity; Niemann-Pick Disease, Type C; Psychotic Disorders	2019
Synthesis and Therapeutic Applications of Iminosugars in Cystic Fibrosis. International journal of molecular sciences, 2020, May-09, Volume: 21, Issue:9 Topics: 1-Deoxynojirimycin; Anti-Inflammatory Agents; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Glycoside Hydrolases; Glycosyltransferases; Heterocyclic Compounds, 1-Ring; Humans; Imino Pyranoses; Inflammation; Molecular Structure; Mutation; Sequence Deletion; Tartrates	2020
GM2 Gangliosidoses: Clinical Features, Pathophysiological Aspects, and Current Therapies. International journal of molecular sciences, 2020, Aug-27, Volume: 21, Issue:17 Topics: 1-Deoxynojirimycin; beta-N-Acetylhexosaminidases; Blood-Brain Barrier; Clinical Trials as Topic; Diet, Ketogenic; G(M2) Activator Protein; G(M2) Ganglioside; Gangliosidoses, GM2; Genetic Therapy; Humans; Mutation; Pyrimethamine; Stem Cell Transplantation	2020
A Systematic Review and Meta-analyses of Longitudinal Studies on Drug Treatments for Gaucher Disease. The Annals of pharmacotherapy, 2023, Volume: 57, Issue:3 Topics: 1-Deoxynojirimycin; Blood Platelets; Enzyme Replacement Therapy; Gaucher Disease; Glucosylceramidase; Humans	2023
Cipaglucosidase Alfa: First Approval. Drugs, 2023, Volume: 83, Issue:8 Topics: 1-Deoxynojirimycin; Adult; Enzyme Replacement Therapy; Glycogen; Glycogen Storage Disease Type II; Humans	2023
Efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: A systematic review. European journal of neurology, 2023, Volume: 30, Issue:9 Topics: 1-Deoxynojirimycin; Adult; Gangliosidoses, GM2; Humans	2023

Trials

26 trial(s) available for 1-deoxynojirimycin and miglustat

Article	Year
Species dependent esterase activities for hydrolysis of an anti-HIV prodrug glycovir and bioavailability of active SC-48334. Pharmaceutical research, 1995, Volume: 12, Issue:8 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Animals; Antiviral Agents; Biological Availability; Butyrates; Dogs; Esterases; Female; HIV; HIV Seropositivity; Humans; Hydrolysis; In Vitro Techniques; Intestinal Absorption; Intestinal Mucosa; Liver; Macaca fascicularis; Male; Piperidines; Prodrugs; Rats; Species Specificity; Subcellular Fractions	1995
The safety and efficacy of combination N-butyl-deoxynojirimycin (SC-48334) and zidovudine in patients with HIV-1 infection and 200-500 CD4 cells/mm3. Journal of acquired immune deficiency syndromes, 1994, Volume: 7, Issue:2 Topics: 1-Deoxynojirimycin; Adult; Antiviral Agents; beta 2-Microglobulin; CD4-Positive T-Lymphocytes; Diarrhea; Double-Blind Method; Drug Therapy, Combination; Female; Follow-Up Studies; Hemoglobins; HIV Core Protein p24; HIV Infections; HIV-1; Humans; Leukocyte Count; Male; Neutrophils; Paresthesia; RNA, Viral; Zidovudine	1994
The tolerability and pharmacokinetics of N-butyl-deoxynojirimycin in patients with advanced HIV disease (ACTG 100). The AIDS Clinical Trials Group (ACTG) of the National Institute of Allergy and Infectious Diseases. Journal of acquired immune deficiency syndromes and human retrovirology : official publication of the International Retrovirology Association, 1995, Dec-15, Volume: 10, Issue:5 Topics: 1-Deoxynojirimycin; Adult; Animals; Antiviral Agents; Cataract; CD4 Lymphocyte Count; CD4-Positive T-Lymphocytes; Diarrhea; Female; Flatulence; Half-Life; HIV Core Protein p24; HIV Infections; HIV-1; Humans; Lens, Crystalline; Leukopenia; Liver; Male; Neutropenia; Rats	1995
Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Lancet (London, England), 2000, Apr-29, Volume: 355, Issue:9214 Topics: 1-Deoxynojirimycin; Administration, Oral; Adult; Aged; Diarrhea; Enzyme Inhibitors; Female; Gaucher Disease; Glucosyltransferases; Half-Life; Hexosaminidases; Humans; Liver; Magnetic Resonance Imaging; Male; Middle Aged; Spleen; Tomography, X-Ray Computed	2000
Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease. Journal of inherited metabolic disease, 2004, Volume: 27, Issue:6 Topics: 1-Deoxynojirimycin; Administration, Oral; Electromyography; Enzyme Inhibitors; Gaucher Disease; Hemoglobins; Hexosaminidases; Humans; Liver; Magnetic Resonance Imaging; Neural Conduction; Platelet Count; Spleen; Tomography, X-Ray Computed	2004
Computerized cognitive testing in patients with type I Gaucher disease: effects of enzyme replacement and substrate reduction. Genetics in medicine : official journal of the American College of Medical Genetics, 2005, Volume: 7, Issue:2 Topics: 1-Deoxynojirimycin; Adult; Aged; Cognition Disorders; Enzyme Inhibitors; Female; Gaucher Disease; Humans; Intelligence Tests; Male; Middle Aged; Neuropsychological Tests; Observer Variation; Psychometrics; Reproducibility of Results; User-Computer Interface	2005
An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment. Clinical therapeutics, 2005, Volume: 27, Issue:8 Topics: 1-Deoxynojirimycin; Adult; Blood; Body Weight; Drug Tolerance; Enzyme Inhibitors; Female; Gaucher Disease; Humans; Liver; Male; Middle Aged; Spleen; Treatment Outcome	2005
Short-term effect of miglustat in every day clinical use in treatment-naïve or previously treated patients with type 1 Gaucher's disease. Haematologica, 2006, Volume: 91, Issue:5 Topics: 1-Deoxynojirimycin; Administration, Oral; Adult; Aged; Bone Marrow; Chemokines, CC; Drug Administration Schedule; Female; Gaucher Disease; Genotype; Glucosylceramidase; Glucosylceramides; Glucosyltransferases; Hexosaminidases; Humans; Male; Middle Aged; Neuropsychological Tests; Quality of Life; Treatment Outcome	2006
[Clinical study of the French cohort of Gaucher disease patients]. La Revue de medecine interne, 2006, Volume: 27 Suppl 1 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Aged, 80 and over; Asthenia; Biomarkers; Child; Child, Preschool; Cohort Studies; Enzyme Inhibitors; Follow-Up Studies; Forecasting; France; Gaucher Disease; Glucosylceramidase; Humans; Infant; Infant, Newborn; Middle Aged; Recombinant Proteins; Splenectomy; Splenomegaly; Time Factors; Treatment Outcome	2006
Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement. Blood, 2007, Oct-01, Volume: 110, Issue:7 Topics: 1-Deoxynojirimycin; Administration, Oral; Adolescent; Adult; Aged; Drug-Related Side Effects and Adverse Reactions; Female; Gaucher Disease; Glucosylceramidase; Humans; Injections, Intravenous; Male; Middle Aged; Quality of Life; Time Factors	2007
Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. The Lancet. Neurology, 2007, Volume: 6, Issue:9 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Age Factors; Analysis of Variance; Child; Child, Preschool; Cohort Studies; Deglutition; Double-Blind Method; Drug Administration Schedule; Enzyme Inhibitors; Female; Humans; Male; Niemann-Pick Disease, Type C; Retrospective Studies; Saccades; Severity of Illness Index	2007
Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. Journal of clinical pharmacology, 2007, Volume: 47, Issue:10 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Area Under Curve; Cross-Over Studies; Female; Food-Drug Interactions; Glucosyltransferases; Half-Life; Humans; Male; Middle Aged; Sex Factors	2007
Randomized, controlled trial of miglustat in Gaucher's disease type 3. Annals of neurology, 2008, Volume: 64, Issue:5 Topics: 1-Deoxynojirimycin; Adolescent; Child; Child, Preschool; Endpoint Determination; Enzyme Inhibitors; Female; Gaucher Disease; Glucosylceramidase; Hexosaminidases; Humans; Liver; Lung; Lung Diseases; Male; Ocular Motility Disorders; Saccades; Spleen; Treatment Outcome; Young Adult	2008
Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment. Genetics in medicine : official journal of the American College of Medical Genetics, 2009, Volume: 11, Issue:6 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Age of Onset; Diarrhea; Enzyme Inhibitors; Fatigue; Female; Glycoside Hydrolase Inhibitors; Humans; Male; Middle Aged; Tay-Sachs Disease; Time Factors; Treatment Outcome; Weight Loss; Young Adult	2009
Pharmacokinetics, safety and tolerability of miglustat in the treatment of pediatric patients with GM2 gangliosidosis. Molecular genetics and metabolism, 2009, Volume: 97, Issue:4 Topics: 1-Deoxynojirimycin; Administration, Oral; Adolescent; Adult; Child; Child, Preschool; Diarrhea; Drug-Related Side Effects and Adverse Reactions; Enzyme Inhibitors; Gangliosidoses, GM2; Glucosyltransferases; Humans; Infant	2009
Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project. Haematologica, 2009, Volume: 94, Issue:12 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Chemokines, CC; Child; Enzyme Inhibitors; Female; Gaucher Disease; Glycoside Hydrolase Inhibitors; Hexosaminidases; Humans; Liver; Male; Middle Aged; Organ Size; Prospective Studies; Quality of Life; Spleen; Surveys and Questionnaires; Time Factors; Treatment Outcome; Weight Loss; Young Adult	2009
Long-term miglustat therapy in children with Niemann-Pick disease type C. Journal of child neurology, 2010, Volume: 25, Issue:3 Topics: 1-Deoxynojirimycin; Child; Child, Preschool; Deglutition; Enzyme Inhibitors; Female; Humans; Male; Motor Activity; Niemann-Pick Disease, Type C; Prospective Studies; Saccades; Time Factors; Treatment Outcome	2010
Changes in the atherogenic profile of patients with type 1 Gaucher disease after miglustat therapy. Atherosclerosis, 2010, Volume: 209, Issue:2 Topics: 1-Deoxynojirimycin; Adult; Aged; Apolipoprotein A-I; Apolipoproteins B; C-Reactive Protein; Cholesterol; Cholesterol, HDL; Cholesterol, LDL; Coronary Disease; Female; Gaucher Disease; Humans; Lipoprotein(a); Male; Middle Aged; Risk; Triglycerides	2010
Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial. Molecular genetics and metabolism, 2010, Volume: 99, Issue:4 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Child; Deglutition; Diarrhea; Female; Humans; Niemann-Pick Disease, Type C; Walking; Weight Loss	2010
Evaluation of miglustat treatment in patients with type III mucopolysaccharidosis: a randomized, double-blind, placebo-controlled study. The Journal of pediatrics, 2011, Volume: 159, Issue:5 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Brain; Child; Child, Preschool; Cognition; Double-Blind Method; Enzyme Inhibitors; Female; Glycoside Hydrolase Inhibitors; Humans; Magnetic Resonance Imaging; Male; Mucopolysaccharidosis III; Neuropsychological Tests; Sleep Wake Disorders	2011
A randomized placebo-controlled trial of miglustat in cystic fibrosis based on nasal potential difference. Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2012, Volume: 11, Issue:3 Topics: 1-Deoxynojirimycin; Administration, Oral; Adolescent; Adult; Child; Cross-Over Studies; Cystic Fibrosis; Dose-Response Relationship, Drug; Double-Blind Method; Enzyme Inhibitors; Female; Follow-Up Studies; Glycoside Hydrolase Inhibitors; Humans; Male; Nasal Mucosa; Sodium Chloride; Treatment Outcome; Young Adult	2012
Miglustat therapy in the French cohort of paediatric patients with Niemann-Pick disease type C. Orphanet journal of rare diseases, 2012, Jun-07, Volume: 7 Topics: 1-Deoxynojirimycin; Adolescent; Age of Onset; Carrier Proteins; Child; Child, Preschool; Cohort Studies; Enzyme Inhibitors; Female; France; Glycoproteins; Humans; Infant; Intracellular Signaling Peptides and Proteins; Male; Membrane Glycoproteins; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Prospective Studies; Treatment Outcome; Vesicular Transport Proteins	2012
Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C. Orphanet journal of rare diseases, 2015, Feb-27, Volume: 10 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Child; Child, Preschool; Drug Administration Schedule; Enzyme Inhibitors; Female; Humans; Infant; Italy; Male; Niemann-Pick Disease, Type C; Young Adult	2015
A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat. Orphanet journal of rare diseases, 2015, Jun-19, Volume: 10 Topics: 1-Deoxynojirimycin; Adult; Cross-Over Studies; Double-Blind Method; Enzyme Inhibitors; Female; Gastrointestinal Tract; Humans; Male; Middle Aged; Placebos; Saccharomyces	2015
Infantile gangliosidoses: Mapping a timeline of clinical changes. Molecular genetics and metabolism, 2017, Volume: 121, Issue:2 Topics: 1-Deoxynojirimycin; Diet, Ketogenic; Disaccharidases; Female; Gangliosidoses; Gangliosidoses, GM2; Gangliosidosis, GM1; Glycoside Hydrolase Inhibitors; Humans; Infant; Male; Prospective Studies; Retrospective Studies	2017
Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial. The Lancet. Neurology, 2021, Volume: 20, Issue:12 Topics: 1-Deoxynojirimycin; Adolescent; alpha-Glucosidases; Double-Blind Method; Glycogen Storage Disease Type II; Humans; Treatment Outcome	2021

Other Studies

256 other study(ies) available for 1-deoxynojirimycin and miglustat

Article	Year
N-alkylated nitrogen-in-the-ring sugars: conformational basis of inhibition of glycosidases and HIV-1 replication. Journal of medicinal chemistry, 1995, Jun-23, Volume: 38, Issue:13 Topics: Alkylation; Animals; Carbohydrate Conformation; Carbohydrate Sequence; Carbohydrates; Cell Line; Glycoside Hydrolases; HIV-1; Intestine, Small; Liver; Magnetic Resonance Spectroscopy; Molecular Sequence Data; Nitrogen; Rats; Virus Replication	1995
Alpha-1-C-alkyl-1-deoxynojirimycin derivatives as potent and selective inhibitors of intestinal isomaltase: remarkable effect of the alkyl chain length on glycosidase inhibitory profile. Bioorganic & medicinal chemistry letters, 2004, Dec-20, Volume: 14, Issue:24 Topics: 1-Deoxynojirimycin; Animals; Evaluation Studies as Topic; Glycoside Hydrolases; Intestine, Small; Molecular Structure; Oligo-1,6-Glucosidase; Rats; Structure-Activity Relationship	2004
Alpha-1-C-octyl-1-deoxynojirimycin as a pharmacological chaperone for Gaucher disease. Bioorganic & medicinal chemistry, 2006, Dec-01, Volume: 14, Issue:23 Topics: 1-Deoxynojirimycin; Cell Line; Dose-Response Relationship, Drug; Gaucher Disease; Glucosamine; Glucosylceramidase; Humans; Molecular Chaperones; Molecular Mimicry; Structure-Activity Relationship	2006
Structural and biochemical evidence for a boat-like transition state in beta-mannosidases. Nature chemical biology, 2008, Volume: 4, Issue:5 Topics: beta-Mannosidase; Enzyme Inhibitors; Hydrolysis; Molecular Mimicry; Protein Conformation	2008
Synthesis and evaluation of D-gluco-pyranocyclopropyl amines as potential glucosidase inhibitors. Bioorganic & medicinal chemistry letters, 2009, Dec-01, Volume: 19, Issue:23 Topics: Amines; Bridged Bicyclo Compounds, Heterocyclic; Drug Evaluation, Preclinical; Enzyme Inhibitors; Glucosidases; Molecular Conformation; Structure-Activity Relationship	2009
Dual-action lipophilic iminosugar improves glycemic control in obese rodents by reduction of visceral glycosphingolipids and buffering of carbohydrate assimilation. Journal of medicinal chemistry, 2010, Jan-28, Volume: 53, Issue:2 Topics: Absorption; Animals; Blood Glucose; Carbohydrate Metabolism; Glycated Hemoglobin; Glycosphingolipids; Imino Sugars; Mice; Mice, Obese; Obesity; Rats; Rats, Zucker; Structure-Activity Relationship; Viscera	2010
Synthesis and α-Glucosidase II inhibitory activity of valienamine pseudodisaccharides relevant to N-glycan biosynthesis. Bioorganic & medicinal chemistry letters, 2011, Sep-15, Volume: 21, Issue:18 Topics: alpha-Glucosidases; Biocatalysis; Chemistry Techniques, Synthetic; Cyclohexenes; Enzyme Inhibitors; Glycoside Hydrolase Inhibitors; Hexosamines; Molecular Conformation; Polysaccharides; Stereoisomerism; Structure-Activity Relationship	2011
α-1-C-butyl-1,4-dideoxy-1,4-imino-l-arabinitol as a second-generation iminosugar-based oral α-glucosidase inhibitor for improving postprandial hyperglycemia. Journal of medicinal chemistry, 2012, Dec-13, Volume: 55, Issue:23 Topics: Administration, Oral; Animals; Enzyme Inhibitors; Glycoside Hydrolase Inhibitors; Humans; Hyperglycemia; Hypoglycemic Agents; Imino Sugars; Inhibitory Concentration 50; Male; Mice; Spectrometry, Mass, Matrix-Assisted Laser Desorption-Ionization	2012
Ester Prodrugs of IHVR-19029 with Enhanced Oral Exposure and Prevention of Gastrointestinal Glucosidase Interaction. ACS medicinal chemistry letters, 2017, Feb-09, Volume: 8, Issue:2 Topics:	2017
5- Journal of medicinal chemistry, 2022, 02-10, Volume: 65, Issue:3 Topics: 1-Deoxynojirimycin; Alkylation; alpha-Glucosidases; Enzyme Inhibitors; Fibroblasts; Glycogen Storage Disease Type II; Humans; Molecular Dynamics Simulation; Molecular Structure; Mutation; Protein Conformation; Protein Stability; Recombinant Proteins	2022
trans, trans-2-C-Aryl-3,4-dihydroxypyrrolidines as potent and selective β-glucosidase inhibitors: Pharmacological chaperones for Gaucher disease. European journal of medicinal chemistry, 2022, Aug-05, Volume: 238 Topics: beta-Glucosidase; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Humans; Molecular Docking Simulation; Pyrrolidines	2022
Modulation of cell-surface transferrin receptor by the imino sugar N-butyldeoxynojirimycin. European journal of biochemistry, 1992, Aug-15, Volume: 208, Issue:1 Topics: 1-Deoxynojirimycin; Antiviral Agents; Cell Line; Cell Membrane; Glucosamine; Glycoside Hydrolases; HIV; Humans; Kinetics; Receptors, Transferrin; Transferrin	1992
Genetic toxicology studies of an anti-AIDS drug. Toxicology letters, 1992, Volume: 60, Issue:3 Topics: 1-Deoxynojirimycin; Acquired Immunodeficiency Syndrome; Animals; Antiviral Agents; Bone Marrow; CHO Cells; Cricetinae; Cricetulus; Drug Evaluation, Preclinical; Female; Glucosamine; Hypoxanthine Phosphoribosyltransferase; Male; Mice; Mice, Inbred Strains; Micronucleus Tests; Salmonella	1992
6-0-butanoylcastanospermine (MDL 28,574) inhibits glycoprotein processing and the growth of HIVs. AIDS (London, England), 1991, Volume: 5, Issue:6 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Cell Line; Cell Line, Transformed; Dose-Response Relationship, Drug; Gene Products, env; Glucosamine; Glycoside Hydrolase Inhibitors; HIV Envelope Protein gp120; HIV Envelope Protein gp160; HIV-1; Humans; Indolizines; Mice; Moloney murine leukemia virus; Protein Precursors; Viral Plaque Assay; Zalcitabine	1991
Inhibition of HIV and SIV infectivity by blockade of alpha-glucosidase activity. Virology, 1991, Volume: 181, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Antiviral Agents; Cell Line; Cell Survival; Dose-Response Relationship, Drug; Glucosamine; Glycoside Hydrolase Inhibitors; HIV; Humans; Kinetics; Models, Biological; Oligosaccharides; Recombination, Genetic; RNA-Directed DNA Polymerase; Simian Immunodeficiency Virus; Vaccinia virus; Viral Envelope Proteins; Viral Plaque Assay; Virus Replication	1991
alpha-Glucosidase II-deficient cells use endo alpha-mannosidase as a bypass route for N-linked oligosaccharide processing. The Journal of biological chemistry, 1991, Feb-25, Volume: 266, Issue:6 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; alpha-Mannosidase; Animals; Chromatography, Affinity; Chromatography, Paper; Glucosamine; Glycoside Hydrolase Inhibitors; Kinetics; Lymphoma; Mannosidases; Mice; Mutation; Oligosaccharides; Tumor Cells, Cultured	1991
Anti-HIV drug mechanism. Nature, 1991, Jul-18, Volume: 352, Issue:6332 Topics: 1-Deoxynojirimycin; Antiviral Agents; Glucosamine; HIV; HIV Envelope Protein gp120; Protein Conformation	1991
Attenuation of HIV-1 infectivity by an inhibitor of oligosaccharide processing. AIDS research and human retroviruses, 1990, Volume: 6, Issue:6 Topics: 1-Deoxynojirimycin; Amino Sugars; Antiviral Agents; Glucosamine; Glucosidases; HIV-1; Humans; Oligosaccharides; Virulence	1990
Several new AIDS drugs being tested. Oncology (Williston Park, N.Y.), 1989, Volume: 3, Issue:6 Topics: 1-Deoxynojirimycin; Acquired Immunodeficiency Syndrome; Animals; Antiviral Agents; CD4 Antigens; Drug Therapy, Combination; Glucosamine; Humans; Interferon Type I; Sarcoma, Kaposi; Zalcitabine; Zidovudine	1989
Experimental treatments for HIV-infected patients. American family physician, 1989, Volume: 39, Issue:6 Topics: 1-Deoxynojirimycin; Antiviral Agents; Glucosamine; HIV Envelope Protein gp160; HIV Seropositivity; Humans; Immunotherapy; Recombinant Proteins; Retroviridae Proteins; Vaccines, Synthetic; Viral Envelope Proteins; Viral Vaccines	1989
Evidence that N-linked glycosylation is necessary for hepatitis B virus secretion. Virology, 1995, Nov-10, Volume: 213, Issue:2 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Base Sequence; DNA Primers; Enzyme Inhibitors; Glycoside Hydrolase Inhibitors; Glycosylation; Hepatitis B Surface Antigens; Hepatitis B virus; Humans; Molecular Sequence Data; Tumor Cells, Cultured; Tunicamycin; Viral Envelope Proteins; Virion	1995
Ligand binding by the immunoglobulin superfamily recognition molecule CD2 is glycosylation-independent. The Journal of biological chemistry, 1995, Jan-06, Volume: 270, Issue:1 Topics: 1-Deoxynojirimycin; Amidohydrolases; Animals; Antibodies, Monoclonal; Antigens, CD; CD2 Antigens; CD58 Antigens; CHO Cells; Cricetinae; Crystallography, X-Ray; Glycosylation; Hexosaminidases; Humans; Immunoglobulins; Ligands; Membrane Glycoproteins; Molecular Structure; Peptide-N4-(N-acetyl-beta-glucosaminyl) Asparagine Amidase	1995
The alpha-glucosidase inhibitor N-butyldeoxynojirimycin inhibits human immunodeficiency virus entry at the level of post-CD4 binding. Journal of virology, 1995, Volume: 69, Issue:9 Topics: 1-Deoxynojirimycin; Antiviral Agents; CD4 Antigens; Cell Line; Glycoside Hydrolase Inhibitors; HIV Envelope Protein gp120; HIV Reverse Transcriptase; HIV-1; Humans; Kinetics; Receptors, Virus; RNA-Directed DNA Polymerase; Time Factors; Virion; Virus Replication	1995
N-butyldeoxygalactonojirimycin inhibits glycolipid biosynthesis but does not affect N-linked oligosaccharide processing. The Journal of biological chemistry, 1994, Oct-28, Volume: 269, Issue:43 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Cells, Cultured; Disease Models, Animal; Gaucher Disease; Glucosylceramidase; Glucosyltransferases; Glycolipids; Humans; Mice; Oligosaccharides; Structure-Activity Relationship	1994
N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis. The Journal of biological chemistry, 1994, Mar-18, Volume: 269, Issue:11 Topics: 1-Deoxynojirimycin; Animals; Cell Line; Gaucher Disease; Glucosyltransferases; Glycolipids; Glycoside Hydrolase Inhibitors; Humans; Lysosomes; Macrophages; Mice; Models, Biological; Structure-Activity Relationship; Tumor Cells, Cultured	1994
Secretion of human hepatitis B virus is inhibited by the imino sugar N-butyldeoxynojirimycin. Proceedings of the National Academy of Sciences of the United States of America, 1994, Mar-15, Volume: 91, Issue:6 Topics: 1-Deoxynojirimycin; Animals; Antiviral Agents; Blotting, Western; Carcinoma, Hepatocellular; Culture Media; DNA, Viral; Hepatitis B Surface Antigens; Hepatitis B virus; Humans; Rabbits; Transfection; Tumor Cells, Cultured; Virion; Virus Replication; Virus Shedding	1994
Mechanism of action of N-butyl deoxynojirimycin in inhibiting HIV-1 infection and activity in combination with nucleoside analogs. AIDS research and human retroviruses, 1993, Volume: 9, Issue:4 Topics: 1-Deoxynojirimycin; Antiviral Agents; CD4 Antigens; Cell Line; Didanosine; Drug Interactions; HIV Envelope Protein gp120; HIV-1; Humans; Nucleosides; Zalcitabine; Zidovudine	1993
Effects of the imino sugar N-butyldeoxynojirimycin on the N-glycosylation of recombinant gp120. The Journal of biological chemistry, 1993, Jan-05, Volume: 268, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Antiviral Agents; Carbon Radioisotopes; CHO Cells; Chromatography, Gel; Cricetinae; Glycosylation; Hexosaminidases; HIV Envelope Protein gp120; HIV-1; Mannose; Mannosyl-Glycoprotein Endo-beta-N-Acetylglucosaminidase; Methionine; Oligosaccharides; Recombinant Proteins; Sulfur Radioisotopes; Transfection; Tritium	1993
Induction of glucosylceramide synthase by synthase inhibitors and ceramide. Biochimica et biophysica acta, 1996, Feb-16, Volume: 1299, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Biological Transport; Cell Membrane; Cells, Cultured; Ceramides; Dogs; Enzyme Inhibitors; Glucosyltransferases; Morpholines; Sphingomyelin Phosphodiesterase	1996
Protease-induced infectivity of hepatitis B virus for a human hepatoblastoma cell line. Journal of virology, 1996, Volume: 70, Issue:4 Topics: 1-Deoxynojirimycin; Amino Acid Sequence; Base Sequence; Chymotrypsin; DNA, Viral; Hepatitis B Antigens; Hepatitis B virus; Hepatoblastoma; Humans; Kinetics; Molecular Sequence Data; Serine Endopeptidases; Tumor Cells, Cultured; Virion	1996
N-butyldeoxynojirimycin-mediated inhibition of human immunodeficiency virus entry correlates with changes in antibody recognition of the V1/V2 region of gp120. Journal of virology, 1996, Volume: 70, Issue:10 Topics: 1-Deoxynojirimycin; Animals; Antiviral Agents; CHO Cells; Cricetinae; Epitopes; HIV Antibodies; HIV Envelope Protein gp120; HIV Infections; HIV-1; Humans; Protein Conformation; Virus Replication	1996
N-butyldeoxynojirimycin-mediated inhibition of human immunodeficiency virus entry correlates with impaired gp120 shedding and gp41 exposure. Journal of virology, 1996, Volume: 70, Issue:10 Topics: 1-Deoxynojirimycin; Animals; Antiviral Agents; CHO Cells; Cricetinae; HIV Envelope Protein gp120; HIV Envelope Protein gp41; HIV Infections; HIV-1; Humans; Virus Replication	1996
Prevention of lysosomal storage in Tay-Sachs mice treated with N-butyldeoxynojirimycin. Science (New York, N.Y.), 1997, Apr-18, Volume: 276, Issue:5311 Topics: 1-Deoxynojirimycin; Animals; Blood-Brain Barrier; Brain; Disease Models, Animal; Enzyme Inhibitors; G(M2) Ganglioside; Lysosomes; Mice; Microscopy, Electron; Neurons; Tay-Sachs Disease	1997
Inhibition of N-glycan processing in B16 melanoma cells results in inactivation of tyrosinase but does not prevent its transport to the melanosome. The Journal of biological chemistry, 1997, Jun-20, Volume: 272, Issue:25 Topics: 1-Deoxynojirimycin; Animals; Biological Transport; Carbohydrate Sequence; Enzyme Inhibitors; Glycosylation; Kinetics; Melanins; Melanocytes; Melanoma; Mice; Molecular Sequence Data; Monophenol Monooxygenase; Oligosaccharides; Polysaccharides; Tumor Cells, Cultured	1997
Sequencing of N-linked oligosaccharides directly from protein gels: in-gel deglycosylation followed by matrix-assisted laser desorption/ionization mass spectrometry and normal-phase high-performance liquid chromatography. Analytical biochemistry, 1997, Jul-15, Volume: 250, Issue:1 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Amidohydrolases; Animals; Antiviral Agents; Carbohydrate Sequence; CHO Cells; Chromatography, High Pressure Liquid; Cricetinae; Electrophoresis, Polyacrylamide Gel; Enzyme Inhibitors; Glycoproteins; Glycoside Hydrolase Inhibitors; Glycoside Hydrolases; HIV Envelope Protein gp120; HIV-1; Humans; Immunoglobulin G; Molecular Sequence Data; Oligosaccharides; Peptide-N4-(N-acetyl-beta-glucosaminyl) Asparagine Amidase; Recombinant Proteins; Sensitivity and Specificity; Spectrometry, Mass, Matrix-Assisted Laser Desorption-Ionization	1997
Extensive glycosphingolipid depletion in the liver and lymphoid organs of mice treated with N-butyldeoxynojirimycin. The Journal of biological chemistry, 1997, Aug-01, Volume: 272, Issue:31 Topics: 1-Deoxynojirimycin; Animals; Body Weight; Cholera Toxin; Enzyme Inhibitors; Female; Glucosyltransferases; Glycosphingolipids; Immunophenotyping; Liver; Lymphoid Tissue; Mice; Mice, Inbred C57BL	1997
The solution NMR structure of glucosylated N-glycans involved in the early stages of glycoprotein biosynthesis and folding. The EMBO journal, 1997, Jul-16, Volume: 16, Issue:14 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Carbohydrate Conformation; Carbohydrate Sequence; CHO Cells; Cricetinae; Enzyme Inhibitors; Glycoproteins; Glycosylation; Magnetic Resonance Spectroscopy; Mannans; Models, Molecular; Molecular Conformation; Molecular Sequence Data; Oligosaccharides; Polysaccharides; Protein Folding	1997
Ultrastructural changes in the Golgi apparatus and secretory granules of HL-60 cells treated with the imino sugar N-butyldeoxynojirimycin. Biology of the cell, 1997, Volume: 89, Issue:2 Topics: 1-Deoxynojirimycin; Cell Membrane; Cytoplasmic Granules; Enzyme Inhibitors; Glucosyltransferases; Glycoside Hydrolase Inhibitors; Golgi Apparatus; HL-60 Cells; Humans; Kinetics; Microscopy, Electron	1997
Differential effects of glycolipid biosynthesis inhibitors on ceramide-induced cell death in neuroblastoma cells. Journal of neurochemistry, 1999, Volume: 72, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Apoptosis; Brain Neoplasms; Cell Death; Cell Division; Ceramides; Enzyme Inhibitors; Gaucher Disease; Glucosylceramides; Glucosyltransferases; Glycolipids; Mice; Morpholines; Neuroblastoma; Rats; Tumor Cells, Cultured	1999
Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N-butyldeoxynojirimycin. Proceedings of the National Academy of Sciences of the United States of America, 1999, May-25, Volume: 96, Issue:11 Topics: 1-Deoxynojirimycin; Aging; Animals; Apoptosis; Behavior, Animal; beta-N-Acetylhexosaminidases; Brain; Dentate Gyrus; Enzyme Inhibitors; Female; Genes, Recessive; Glycoside Hydrolase Inhibitors; Glycosphingolipids; Hexosaminidase B; Life Expectancy; Liver; Mice; Mice, Mutant Strains; Motor Activity; Sandhoff Disease	1999
Effects of N-butyldeoxynojirimycin and the Lec3.2.8.1 mutant phenotype on N-glycan processing in Chinese hamster ovary cells: application to glycoprotein crystallization. Protein science : a publication of the Protein Society, 1999, Volume: 8, Issue:8 Topics: 1-Deoxynojirimycin; Animals; Antigens, CD; CHO Cells; Cricetinae; Cricetulus; Crystallization; Crystallography, X-Ray; Glycoproteins; Humans; Mutation; Phenotype; Polysaccharides; Recombinant Proteins	1999
Protein specific N-glycosylation of tyrosinase and tyrosinase-related protein-1 in B16 mouse melanoma cells. The Biochemical journal, 1999, Dec-15, Volume: 344 Pt 3 Topics: 1-Deoxynojirimycin; Amidohydrolases; Animals; Glycoside Hydrolases; Glycosylation; Hexosaminidases; Membrane Glycoproteins; Mice; Monophenol Monooxygenase; Oligosaccharides; Oxidoreductases; Peptide-N4-(N-acetyl-beta-glucosaminyl) Asparagine Amidase; Polysaccharides; Proteins; Tumor Cells, Cultured	1999
Glucosidase and mannosidase inhibitors mediate increased secretion of mutant alpha1 antitrypsin Z. The Journal of biological chemistry, 2000, Jan-21, Volume: 275, Issue:3 Topics: 1-Deoxynojirimycin; Alkaloids; alpha 1-Antitrypsin; Cell Line; Cell-Free System; Cysteine Proteinase Inhibitors; Enzyme Inhibitors; Glucosidases; Humans; Indolizines; Leupeptins; Mannosidases; Microsomes; Mutagenesis; Time Factors	2000
Investigation of the intracellular transport of tyrosinase and tyrosinase related protein (TRP)-1. The effect of endoplasmic reticulum (ER)-glucosidases inhibition. Cellular and molecular biology (Noisy-le-Grand, France), 1999, Volume: 45, Issue:7 Topics: 1-Deoxynojirimycin; Animals; Biological Transport, Active; Endoplasmic Reticulum; Glucosidases; Glycosylation; Intracellular Fluid; Melanoma, Experimental; Melanosomes; Membrane Glycoproteins; Mice; Microscopy, Electron; Monophenol Monooxygenase; Neoplasm Proteins; Oxidation-Reduction; Oxidoreductases; Protein Conformation; Protein Processing, Post-Translational; Proteins; Tumor Cells, Cultured	1999
Glycosphingolipid depletion in fabry disease lymphoblasts with potent inhibitors of glucosylceramide synthase. Kidney international, 2000, Volume: 57, Issue:2 Topics: 1-Deoxynojirimycin; alpha-Galactosidase; B-Lymphocytes; Bacterial Toxins; Cell Line, Transformed; Dose-Response Relationship, Drug; Enzyme Inhibitors; Fabry Disease; Fluorescein-5-isothiocyanate; Fluorescent Dyes; Genetic Vectors; Glucosyltransferases; Glycosphingolipids; Herpesvirus 4, Human; Humans; Neutral Glycosphingolipids; Propanolamines; Pyrrolidines; Shiga Toxin 1; Trihexosylceramides	2000
Suppressive effects of swainsonine and N-butyldeoxynojirimycin on human bone marrow neutrophil maturation. Biochemical and biophysical research communications, 2000, Mar-05, Volume: 269, Issue:1 Topics: 1-Deoxynojirimycin; alpha-Mannosidase; Apoptosis; Base Sequence; Bone Marrow Cells; CCAAT-Enhancer-Binding Proteins; Cell Differentiation; Cells, Cultured; Colony-Forming Units Assay; DNA Primers; DNA-Binding Proteins; Enzyme Inhibitors; Glycoside Hydrolase Inhibitors; Granulocyte Colony-Stimulating Factor; Hematopoiesis; Humans; Mannosidases; Neutrophils; Nuclear Proteins; Oligosaccharides; Receptors, Granulocyte Colony-Stimulating Factor; RNA, Messenger; Swainsonine; Tunicamycin	2000
N-butyldeoxygalactonojirimycin: a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo. Biochemical pharmacology, 2000, Apr-01, Volume: 59, Issue:7 Topics: 1-Deoxynojirimycin; Animals; Carbon Radioisotopes; Cell Division; Disaccharidases; Enzyme Inhibitors; Female; Glycogen; Glycosphingolipids; Humans; Liver; Lymphocytes; Mice; Mice, Inbred C57BL; Tissue Distribution	2000
Reduction of globotriaosylceramide in Fabry disease mice by substrate deprivation. The Journal of clinical investigation, 2000, Volume: 105, Issue:11 Topics: 1-Deoxynojirimycin; Animals; Dose-Response Relationship, Drug; Fabry Disease; Kidney; Male; Mice; Mice, Inbred C57BL; Mice, Knockout; Propanolamines; Pyrrolidines; Trihexosylceramides	2000
Hepatitis C virus glycoprotein E2 binding to CD81: the role of E1E2 cleavage and protein glycosylation in bioactivity. Virology, 2000, Jul-20, Volume: 273, Issue:1 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Antigens, CD; Cell Line; Glycoside Hydrolase Inhibitors; Glycosylation; Hepacivirus; Humans; Membrane Proteins; Protein Binding; Protein Processing, Post-Translational; Recombinant Fusion Proteins; Sequence Deletion; Spodoptera; Tetraspanin 28; Viral Envelope Proteins	2000
Folding and maturation of tyrosinase-related protein-1 are regulated by the post-translational formation of disulfide bonds and by N-glycan processing. The Journal of biological chemistry, 2000, Oct-13, Volume: 275, Issue:41 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Amidohydrolases; Animals; Calcium-Binding Proteins; Calnexin; Disulfides; Dithiothreitol; Endoplasmic Reticulum; Glycoside Hydrolase Inhibitors; Hexosaminidases; Kinetics; Melanoma; Membrane Glycoproteins; Mice; Molecular Chaperones; Oxidoreductases; Peptide-N4-(N-acetyl-beta-glucosaminyl) Asparagine Amidase; Polysaccharides; Protein Binding; Protein Conformation; Protein Denaturation; Protein Folding; Protein Processing, Post-Translational; Protein Transport; Proteins; Thermodynamics; Tumor Cells, Cultured	2000
High-performance cation-exchange chromatography and pulsed amperometric detection for the separation, detection, and quantitation of N-alkylated imino sugars in biological samples. Analytical biochemistry, 2000, Aug-15, Volume: 284, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Brain; Carbohydrates; Cations; Chromatography, Ion Exchange; Electrochemistry; Liver; Lysosomal Storage Diseases; Mice; Mice, Inbred C57BL; Models, Chemical; Time Factors	2000
Treatment of Gaucher's disease with OGT 918. Lancet (London, England), 2000, Aug-19, Volume: 356, Issue:9230 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Glucosylceramides; Glycoside Hydrolase Inhibitors; Hepatomegaly; Hexosaminidases; Humans; Splenomegaly; Time	2000
Treatment of Gaucher's disease with OGT 918. Lancet (London, England), 2000, Aug-19, Volume: 356, Issue:9230 Topics: 1-Deoxynojirimycin; Administration, Oral; Enzyme Inhibitors; Gaucher Disease; Glycoside Hydrolase Inhibitors; Humans; Randomized Controlled Trials as Topic; Time; Treatment Outcome	2000
Risks of Gaucher's treatment. Lancet (London, England), 2000, Oct-14, Volume: 356, Issue:9238 Topics: 1-Deoxynojirimycin; Anti-HIV Agents; Dose-Response Relationship, Drug; Gaucher Disease; Humans; Risk Factors	2000
Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation. Blood, 2001, Jan-01, Volume: 97, Issue:1 Topics: 1-Deoxynojirimycin; Animals; beta-N-Acetylhexosaminidases; Bone Marrow Transplantation; Brain; Diagnostic Techniques, Neurological; Disease Models, Animal; Enzyme Inhibitors; Glycosphingolipids; Hexosaminidase B; Mice; Sandhoff Disease; Spinal Cord; Survival Rate	2001
Imino sugar therapy for type 1 Gaucher disease. Glycobiology, 2000, Volume: 10, Issue:11 Topics: 1-Deoxynojirimycin; Animals; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Glycosphingolipids; Humans; Mice	2000
Antiviral effect of N-butyldeoxynojirimycin against bovine viral diarrhea virus correlates with misfolding of E2 envelope proteins and impairment of their association into E1-E2 heterodimers. Journal of virology, 2001, Volume: 75, Issue:8 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Antibodies, Monoclonal; Antibodies, Viral; Antiviral Agents; Calcium-Binding Proteins; Calnexin; Calreticulin; Cell Line; Diarrhea Viruses, Bovine Viral; Dimerization; Disulfides; Dogs; Glycoproteins; Glycoside Hydrolase Inhibitors; Hepacivirus; Kinetics; Protein Binding; Protein Conformation; Protein Folding; Protein Processing, Post-Translational; Ribonucleoproteins; Viral Envelope Proteins; Viral Plaque Assay	2001
N -butyldeoxynojirimycin reduces growth and ganglioside content of experimental mouse brain tumours. British journal of cancer, 2001, Apr-20, Volume: 84, Issue:8 Topics: 1-Deoxynojirimycin; Animals; Body Weight; Brain; Brain Neoplasms; Cell Count; Cell Division; Gangliosides; Intestines; Liver; Male; Methylcholanthrene; Mice; Mice, Inbred C57BL; Time Factors; Tissue Distribution; Tumor Cells, Cultured	2001
Critical role for glycosphingolipids in Niemann-Pick disease type C. Current biology : CB, 2001, Aug-21, Volume: 11, Issue:16 Topics: 1-Deoxynojirimycin; Age Factors; Animals; Carrier Proteins; Cats; Cerebellum; Disease Models, Animal; Enzyme Inhibitors; Glycoside Hydrolase Inhibitors; Glycosphingolipids; Humans; Immunohistochemistry; Intracellular Signaling Peptides and Proteins; Membrane Glycoproteins; Mice; Niemann-Pick C1 Protein; Niemann-Pick Diseases	2001
Inhibition of host ER glucosidase activity prevents Golgi processing of virion-associated bovine viral diarrhea virus E2 glycoproteins and reduces infectivity of secreted virions. Virology, 2002, Mar-30, Volume: 295, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Bovine Virus Diarrhea-Mucosal Disease; Cattle; Cell Line; Diarrhea Viruses, Bovine Viral; Endoplasmic Reticulum; Enzyme Inhibitors; Glucosidases; Glycoproteins; Glycoside Hydrolases; Golgi Apparatus; Hexosaminidases; Mannosyl-Glycoprotein Endo-beta-N-Acetylglucosaminidase; Viral Envelope Proteins; Virion; Virus Replication	2002
pH-sensitive liposomes are efficient carriers for endoplasmic reticulum-targeted drugs in mouse melanoma cells. Biochemical and biophysical research communications, 2002, May-10, Volume: 293, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Dose-Response Relationship, Drug; Drug Delivery Systems; Endoplasmic Reticulum; Enzyme Inhibitors; Hydrogen-Ion Concentration; Kinetics; Liposomes; Melanoma; Mice; Monophenol Monooxygenase; Pigmentation; Tumor Cells, Cultured	2002
The combination of interferon alpha-2b and n-butyl deoxynojirimycin has a greater than additive antiviral effect upon production of infectious bovine viral diarrhea virus (BVDV) in vitro: implications for hepatitis C virus (HCV) therapy. Antiviral research, 2002, Volume: 55, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Antiviral Agents; Cattle; Diarrhea Viruses, Bovine Viral; Dose-Response Relationship, Drug; Drug Design; Drug Synergism; Drug Therapy, Combination; Hepacivirus; Hepatitis C; Humans; In Vitro Techniques; Interferon-alpha; Regression Analysis; Tumor Cells, Cultured; Viral Plaque Assay	2002
Calnexin, calreticulin, and ERp57 cooperate in disulfide bond formation in human CD1d heavy chain. The Journal of biological chemistry, 2002, Nov-22, Volume: 277, Issue:47 Topics: 1-Deoxynojirimycin; Animals; Antigens, CD1; Antigens, CD1d; Calnexin; Calreticulin; Cell Line; Disulfides; Endoplasmic Reticulum; Glucose; Glycoside Hydrolase Inhibitors; Heat-Shock Proteins; Humans; Indolizines; Isomerases; Molecular Chaperones; Oxidation-Reduction; Protein Disulfide-Isomerases; Protein Folding; Protein Processing, Post-Translational; Protein Transport	2002
Reversible infertility in male mice after oral administration of alkylated imino sugars: a nonhormonal approach to male contraception. Proceedings of the National Academy of Sciences of the United States of America, 2002, Dec-24, Volume: 99, Issue:26 Topics: 1-Deoxynojirimycin; Administration, Oral; Animals; Contraceptive Agents, Male; Dose-Response Relationship, Drug; Female; Fertility; Male; Mice; Mice, Inbred C57BL; Organ Size; Sperm Count; Testis; Testosterone	2002
Glycolipid depletion in antimicrobial therapy. Molecular microbiology, 2003, Volume: 47, Issue:2 Topics: 1-Deoxynojirimycin; Animals; Bacterial Adhesion; Cell Line; Enzyme Inhibitors; Escherichia coli; Escherichia coli Infections; Fimbriae, Bacterial; Glycosphingolipids; Humans; Mice; Mice, Inbred C3H; Receptors, Cell Surface; Recombinant Proteins; Urinary Tract Infections	2003
Central nervous system inflammation is a hallmark of pathogenesis in mouse models of GM1 and GM2 gangliosidosis. Brain : a journal of neurology, 2003, Volume: 126, Issue:Pt 4 Topics: 1-Deoxynojirimycin; Animals; Antigens, CD; Apoptosis; Biomarkers; Blood-Brain Barrier; Cytokines; Enzyme Inhibitors; Gangliosidoses; Gangliosidoses, GM2; Gangliosidosis, GM1; Genes, MHC Class II; Immunohistochemistry; Inflammation; Mice; Sandhoff Disease; Tay-Sachs Disease	2003
[Drug for Gaucher disease also works as sperm inhibitor. Is the "pill" for the man on its way?]. MMW Fortschritte der Medizin, 2003, Feb-13, Volume: 145, Issue:7 Topics: 1-Deoxynojirimycin; Animals; Contraceptive Agents, Male; Drug Implants; Enzyme Inhibitors; Female; Gaucher Disease; Glycoside Hydrolase Inhibitors; Humans; Injections, Subcutaneous; Male; Mice; Pregnancy; Spermatogenesis	2003
Inhibition of calcium uptake via the sarco/endoplasmic reticulum Ca2+-ATPase in a mouse model of Sandhoff disease and prevention by treatment with N-butyldeoxynojirimycin. The Journal of biological chemistry, 2003, Aug-08, Volume: 278, Issue:32 Topics: 1-Deoxynojirimycin; Adenosine Triphosphate; Animals; Blotting, Western; Brain; Calcium; Calcium-Transporting ATPases; Cell Death; Disease Models, Animal; Dose-Response Relationship, Drug; Electrophoresis, Polyacrylamide Gel; Endoplasmic Reticulum; Enzyme Inhibitors; G(M2) Ganglioside; Gangliosides; Genotype; Glycolipids; Hippocampus; Kinetics; Lipid Metabolism; Mice; Mice, Transgenic; Microsomes; Neurons; Reverse Transcriptase Polymerase Chain Reaction; Sandhoff Disease; Sarcoplasmic Reticulum; Sarcoplasmic Reticulum Calcium-Transporting ATPases; Spectrophotometry; Thapsigargin; Time Factors	2003
Epitope-dependent blocking of the angiotensin-converting enzyme dimerization by monoclonal antibodies to the N-terminal domain of ACE: possible link of ACE dimerization and shedding from the cell surface. Biochemistry, 2003, Jun-17, Volume: 42, Issue:23 Topics: 1-Deoxynojirimycin; Animals; Antibodies, Monoclonal; Antibody Specificity; Carbohydrate Metabolism; Carbohydrates; Cattle; Cell Membrane; Cells, Cultured; CHO Cells; Cricetinae; Cross-Linking Reagents; Dimerization; Epitopes; Glucosidases; Humans; Micelles; Peptidyl-Dipeptidase A; Protein Structure, Tertiary; Water	2003
N-butyldeoxynojirimycin inhibits murine melanoma cell ganglioside metabolism and delays tumor onset. Cancer letters, 2003, Nov-10, Volume: 201, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Cell Division; Cell Line, Tumor; Ceramides; Disease Progression; Dose-Response Relationship, Drug; Enzyme Inhibitors; Gangliosides; Glucosyltransferases; Melanoma, Experimental; Mice; Neoplasm Transplantation	2003
Ceramide synthesis correlates with the posttranscriptional regulation of the sterol-regulatory element-binding protein. Arteriosclerosis, thrombosis, and vascular biology, 2004, Volume: 24, Issue:5 Topics: 1-Deoxynojirimycin; Acyltransferases; Amidohydrolases; Animals; CCAAT-Enhancer-Binding Proteins; Ceramides; CHO Cells; Cholesterol; Cricetinae; Cricetulus; Cycloserine; DNA-Binding Proteins; Enzyme Induction; Fatty Acids, Monounsaturated; Fumonisins; Genes, Reporter; Hydroxymethylglutaryl-CoA Synthase; Morpholines; Myristates; Phosphotransferases (Alcohol Group Acceptor); Propanolamines; RNA Processing, Post-Transcriptional; Serine C-Palmitoyltransferase; Sphingolipids; Sphingosine; Sterol Regulatory Element Binding Protein 1; Transcription Factors; Transcription, Genetic; Transfection	2004
Inhibition of alpha-glucosidases I and II increases the cell surface expression of functional class A macrophage scavenger receptor (SR-A) by extending its half-life. The Journal of biological chemistry, 2004, Sep-17, Volume: 279, Issue:38 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Antineoplastic Agents; Calnexin; Carbocyanines; Drug Synergism; Electrophoretic Mobility Shift Assay; Endocytosis; Enzyme Inhibitors; Glycoside Hydrolase Inhibitors; Glycosylation; Interferon-gamma; Lipoproteins, LDL; Macrophages; Mice; Protein Transport; Receptors, Cell Surface; Receptors, Immunologic; Receptors, Scavenger; Reverse Transcriptase Polymerase Chain Reaction; RNA, Messenger; Scavenger Receptors, Class A	2004
Alkylated imino sugars, reversible male infertility-inducing agents, do not affect the genetic integrity of male mouse germ cells during short-term treatment despite induction of sperm deformities. Biology of reproduction, 2005, Volume: 72, Issue:4 Topics: 1-Deoxynojirimycin; Alkylation; Animals; Carbohydrates; Contraceptive Agents, Male; Disulfides; Female; Fertility; Fertilization in Vitro; Imines; Infertility, Male; Male; Mice; Mice, Inbred C57BL; Mice, Inbred DBA; Pregnancy; Pregnancy Outcome; Protamines; Spermatozoa; Sphingolipids	2005
Glycosphingolipid accumulation inhibits cholesterol efflux via the ABCA1/apolipoprotein A-I pathway: 1-phenyl-2-decanoylamino-3-morpholino-1-propanol is a novel cholesterol efflux accelerator. The Journal of biological chemistry, 2005, Jul-01, Volume: 280, Issue:26 Topics: 1-Deoxynojirimycin; Antigens, CD; Apolipoprotein A-I; ATP Binding Cassette Transporter 1; ATP-Binding Cassette Transporters; Biotinylation; Blotting, Western; Cell Line; Cell Membrane; Cholesterol; Chromatography, High Pressure Liquid; Dose-Response Relationship, Drug; Enzyme Inhibitors; Fibroblasts; Glycosphingolipids; Humans; Lactosylceramides; Lipoproteins, LDL; Monocytes; Morpholines; Phospholipids; Reverse Transcriptase Polymerase Chain Reaction; RNA, Messenger; Time Factors; Up-Regulation	2005
Allogeneic BMT followed by substrate reduction therapy in a child with subacute Tay-Sachs disease. Bone marrow transplantation, 2005, Volume: 36, Issue:10 Topics: 1-Deoxynojirimycin; Bone Marrow Transplantation; Child; Female; Humans; Nervous System Diseases; Tay-Sachs Disease; Transplantation, Homologous	2005
Long-term non-hormonal male contraception in mice using N-butyldeoxynojirimycin. Human reproduction (Oxford, England), 2006, Volume: 21, Issue:5 Topics: 1-Deoxynojirimycin; Animals; Behavior, Animal; Contraception; Contraceptive Agents, Male; Fertility; Follicle Stimulating Hormone; Imino Sugars; Luteinizing Hormone; Male; Mice; Sperm Count; Sperm Motility; Spermatozoa; Testis; Testosterone	2006
Substrate reduction therapy in the infantile form of Tay-Sachs disease. Neurology, 2006, Jan-24, Volume: 66, Issue:2 Topics: 1-Deoxynojirimycin; Craniofacial Abnormalities; Electroencephalography; Evoked Potentials, Auditory, Brain Stem; Evoked Potentials, Visual; Female; Humans; Infant; Nerve Degeneration; Tay-Sachs Disease	2006
Rescue of functional delF508-CFTR channels in cystic fibrosis epithelial cells by the alpha-glucosidase inhibitor miglustat. FEBS letters, 2006, Apr-03, Volume: 580, Issue:8 Topics: 1-Deoxynojirimycin; Animals; Calnexin; Chlorocebus aethiops; COS Cells; Cricetinae; Cricetulus; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Glycoside Hydrolase Inhibitors; Humans; In Vitro Techniques; Intestinal Mucosa; Mice; Mice, Inbred CFTR; Mice, Knockout; Protein Binding; Protein Processing, Post-Translational; Protein Transport	2006
Gangliosides do not affect ABC transporter function in human neuroblastoma cells. Journal of lipid research, 2006, Volume: 47, Issue:6 Topics: 1-Deoxynojirimycin; ATP Binding Cassette Transporter, Subfamily B, Member 1; ATP-Binding Cassette Transporters; Cell Line, Tumor; Ceramides; Enzyme Inhibitors; Gangliosides; Glucosyltransferases; Humans; Immunoblotting; Neuroblastoma	2006
D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol alters cellular cholesterol homeostasis by modulating the endosome lipid domains. Biochemistry, 2006, Apr-11, Volume: 45, Issue:14 Topics: 1-Deoxynojirimycin; 4-Chloro-7-nitrobenzofurazan; Cells, Cultured; Ceramides; Cholesterol; Endosomes; Enzyme Activation; Fibroblasts; Glucosyltransferases; Homeostasis; Humans; Lipase; Lysophospholipids; Membrane Microdomains; Monoglycerides; Morpholines; Neuroblastoma; Paclitaxel; Stereoisomerism; Sterol Esterase	2006
Therapeutic goals in Gaucher disease. La Revue de medecine interne, 2006, Volume: 27 Suppl 1 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Age Factors; Child; Evidence-Based Medicine; Gaucher Disease; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Humans; Injections, Intravenous; Phenotype; Quality of Life; Recombinant Proteins; Splenectomy; Splenomegaly; Time Factors	2006
Inhibition of hybrid- and complex-type glycosylation reveals the presence of the GlcNAc transferase I-independent fucosylation pathway. Glycobiology, 2006, Volume: 16, Issue:8 Topics: 1-Deoxynojirimycin; Alkaloids; alpha-Mannosidase; Animals; Cell Line; CHO Cells; Cricetinae; DNA, Complementary; Enzyme Inhibitors; Fucose; Glycoproteins; Glycosylation; Humans; N-Acetylglucosaminyltransferases; Recombinant Proteins; Spectrometry, Mass, Electrospray Ionization; Spectrometry, Mass, Matrix-Assisted Laser Desorption-Ionization; Swainsonine	2006
Synthesis and evaluation of glycosidase inhibitory activity of N-butyl 1-deoxy-D-gluco-homonojirimycin and N-butyl 1-deoxy-L-ido-homonojirimycin. Bioorganic & medicinal chemistry, 2006, Aug-15, Volume: 14, Issue:16 Topics: 1-Deoxynojirimycin; Amination; Enzyme Inhibitors; Glycoside Hydrolases; Imino Pyranoses; Inhibitory Concentration 50; Models, Chemical; Piperidines	2006
Effective treatment of an elderly patient with Gaucher's disease and Parkinsonism: a case report of 24 months' oral substrate reduction therapy with miglustat. Parkinsonism & related disorders, 2007, Volume: 13, Issue:6 Topics: 1-Deoxynojirimycin; Administration, Oral; Aged; Enzyme Inhibitors; Follow-Up Studies; Gaucher Disease; Hexosaminidases; Humans; Male; Parkinson Disease; Peptidyl-Dipeptidase A	2007
Miglustat has no apparent effect on spermatogenesis in normal men. Human reproduction (Oxford, England), 2007, Volume: 22, Issue:3 Topics: 1-Deoxynojirimycin; Acrosome Reaction; Adult; Humans; Male; Pilot Projects; Semen; Sperm Count; Sperm Motility; Spermatogenesis; Testosterone	2007
Severe endothelial dysfunction in the aorta of a mouse model of Fabry disease; partial prevention by N-butyldeoxynojirimycin treatment. Journal of inherited metabolic disease, 2007, Volume: 30, Issue:1 Topics: 1-Deoxynojirimycin; alpha-Galactosidase; Animals; Aorta; Disease Models, Animal; Enzyme Inhibitors; Fabry Disease; Female; Humans; Male; Mice; Mice, Inbred C57BL; Mice, Knockout; Microscopy, Electron; Phenotype	2007
Selective action of the iminosugar isofagomine, a pharmacological chaperone for mutant forms of acid-beta-glucosidase. Biochemical pharmacology, 2007, May-01, Volume: 73, Issue:9 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Cell Proliferation; Cells, Cultured; Disaccharidases; Gaucher Disease; Glucosylceramidase; Glucosyltransferases; Glycoside Hydrolases; Humans; Imino Pyranoses; Lysosomes; Mutation; Oligosaccharides; Piperidines	2007
The sensitivity of murine spermiogenesis to miglustat is a quantitative trait: a pharmacogenetic study. Reproductive biology and endocrinology : RB&E, 2007, Jan-22, Volume: 5 Topics: 1-Deoxynojirimycin; Acrosome; Animals; Drug Resistance; Enzyme Inhibitors; Female; Infertility, Male; Male; Mice; Mice, Inbred AKR; Mice, Inbred BALB C; Mice, Inbred C3H; Mice, Inbred C57BL; Mice, Inbred CBA; Mice, Inbred DBA; Mice, Inbred MRL lpr; Mice, Inbred NZB; Pregnancy; Quantitative Trait, Heritable; Rabbits; Sexual Behavior, Animal; Species Specificity; Sperm Motility; Spermatogenesis	2007
Treating patients with Gaucher disease and parkinsonism: misrepresentation in a title. Parkinsonism & related disorders, 2008, Volume: 14, Issue:1 Topics: 1-Deoxynojirimycin; Aged; Enzyme Inhibitors; Gaucher Disease; Humans; Male; Parkinsonian Disorders	2008
Neurologic improvement in a type 3 Gaucher disease patient treated with imiglucerase/miglustat combination. Epilepsia, 2007, Volume: 48, Issue:7 Topics: 1-Deoxynojirimycin; Adult; Comorbidity; Disease Progression; Drug Administration Schedule; Drug Therapy, Combination; Dystonia; Electroencephalography; Enzyme Inhibitors; Epilepsies, Myoclonic; Gaucher Disease; Glucosylceramidase; Humans; Male; Nervous System Diseases; Recombinant Proteins; Syndrome; Treatment Outcome	2007
Differential alteration of lipid antigen presentation to NKT cells due to imbalances in lipid metabolism. European journal of immunology, 2007, Volume: 37, Issue:6 Topics: 1-Deoxynojirimycin; Animals; Antigen Presentation; Antigens, CD1; Antigens, CD1d; beta-Galactosidase; CD4 Antigens; Dendritic Cells; Enzyme Inhibitors; Galactosylceramides; Globosides; Glycolipids; Humans; Lipid Metabolism; Lipid Metabolism Disorders; Liver; Mice; Mice, Inbred BALB C; Mice, Inbred C57BL; Mice, Knockout; Receptors, Antigen, T-Cell; Spleen; T-Lymphocyte Subsets; Thymus Gland; Trihexosylceramides; Vesicular Transport Proteins	2007
Treatment of hepatitis B virus-infected cells with alpha-glucosidase inhibitors results in production of virions with altered molecular composition and infectivity. Antiviral research, 2007, Volume: 76, Issue:1 Topics: 1-Deoxynojirimycin; Cell Line; Endoplasmic Reticulum; Enzyme Inhibitors; Glycoside Hydrolase Inhibitors; Glycosylation; Hepatitis B; Hepatitis B virus; Humans; Viral Envelope Proteins; Virulence; Virus Replication	2007
Calcium homeostasis is abnormal in cystic fibrosis airway epithelial cells but is normalized after rescue of F508del-CFTR. Cell calcium, 2008, Volume: 43, Issue:2 Topics: 1-Deoxynojirimycin; Adenosine Triphosphate; Benzoates; Boron Compounds; Calcium Signaling; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Endoplasmic Reticulum; Epithelial Cells; Histamine; Humans; Inositol 1,4,5-Trisphosphate Receptors; Nocodazole; Protein Transport; Respiratory Mucosa; Sequence Deletion; Temperature; Thiazolidines	2008
Treatment of Niemann-Pick disease type C in two children with miglustat: initial responses and maintenance of effects over 1 year. Journal of inherited metabolic disease, 2007, Volume: 30, Issue:5 Topics: 1-Deoxynojirimycin; Adolescent; Child; Cognition; Deglutition; Enzyme Inhibitors; Glucosyltransferases; Humans; Interpersonal Relations; Niemann-Pick Disease, Type C; Recovery of Function; Severity of Illness Index; Time Factors; Treatment Outcome; Verbal Behavior; Walking	2007
The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat. Xenobiotica; the fate of foreign compounds in biological systems, 2007, Volume: 37, Issue:3 Topics: 1-Deoxynojirimycin; Administration, Oral; Animals; Biological Availability; Carbon Radioisotopes; Enzyme Inhibitors; Female; Glucosyltransferases; Injections, Intravenous; Male; Prodrugs; Rats; Rats, Sprague-Dawley; Tissue Distribution; Tritium	2007
Crystal structures of complexes of N-butyl- and N-nonyl-deoxynojirimycin bound to acid beta-glucosidase: insights into the mechanism of chemical chaperone action in Gaucher disease. The Journal of biological chemistry, 2007, Sep-28, Volume: 282, Issue:39 Topics: 1-Deoxynojirimycin; Administration, Oral; Binding Sites; Crystallography, X-Ray; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Glucosylceramides; Humans; Hydrophobic and Hydrophilic Interactions; Molecular Chaperones; Recombinant Proteins; Structure-Activity Relationship	2007
[Niemann-Pick diseases in adults]. La Revue de medecine interne, 2007, Volume: 28 Suppl 4 Topics: 1-Deoxynojirimycin; Adult; Carrier Proteins; Enzyme Inhibitors; Glycoproteins; Glycoside Hydrolase Inhibitors; Humans; Intracellular Signaling Peptides and Proteins; Membrane Glycoproteins; Middle Aged; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type B; Niemann-Pick Disease, Type C; Sphingomyelin Phosphodiesterase; Vesicular Transport Proteins	2007
[New data on Gaucher's disease]. La Revue de medecine interne, 2008, Volume: 29, Issue:3 Topics: 1-Deoxynojirimycin; Clinical Protocols; Clinical Trials, Phase I as Topic; Clinical Trials, Phase II as Topic; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Humans; Phenotype; Recombinant Proteins	2008
N-butyldeoxynojirimycin causes weight loss as a result of appetite suppression in lean and obese mice. Diabetes, obesity & metabolism, 2008, Volume: 10, Issue:2 Topics: 1-Deoxynojirimycin; Adipose Tissue; Animals; Appetite Regulation; Enzyme Inhibitors; Leptin; Mice; Mice, Obese; Obesity; Weight Loss	2008
[Organization of Gaucher disease management in France]. La Revue de medecine interne, 2007, Volume: 28 Suppl 2 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Child; Child, Preschool; Enzyme Inhibitors; Female; France; Gaucher Disease; Glucosylceramidase; Hospitalization; Humans; Infant; Infant, Newborn; Male; Middle Aged; Parkinsonian Disorders; Registries; Saposins; Treatment Outcome	2007
Parallel improvement of sodium and chloride transport defects by miglustat (n-butyldeoxynojyrimicin) in cystic fibrosis epithelial cells. The Journal of pharmacology and experimental therapeutics, 2008, Volume: 325, Issue:3 Topics: 1-Deoxynojirimycin; Amiloride; Animals; Cell Line; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Humans; Mice; Mice, Knockout; Nasal Mucosa; Sodium; Sodium Channel Blockers	2008
Gaucher disease: improving management. Acta paediatrica (Oslo, Norway : 1992), 2008, Volume: 97, Issue:457 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Gaucher Disease; Glycoside Hydrolase Inhibitors; Humans	2008
Splenic nodules in paediatric Gaucher disease treated by enzyme replacement therapy. Pediatric radiology, 2008, Volume: 38, Issue:6 Topics: 1-Deoxynojirimycin; Child; Child, Preschool; Cohort Studies; Enzyme Inhibitors; Female; Follow-Up Studies; Gaucher Disease; Glucosylceramidase; Humans; Infant; Male; Retrospective Studies; Spleen; Splenic Diseases; Splenomegaly; Ultrasonography	2008
Beneficial effects of substrate reduction therapy in a mouse model of GM1 gangliosidosis. Molecular genetics and metabolism, 2008, Volume: 94, Issue:2 Topics: 1-Deoxynojirimycin; Animals; beta-Galactosidase; Brain; Disease Models, Animal; Enzyme Inhibitors; Feces; Gangliosidosis, GM1; Glucosyltransferases; Glycosphingolipids; Humans; Macrophage Activation; Mice; Mice, Knockout; Motor Activity; Protein Transport	2008
Changes of bone metabolism in seven patients with Gaucher disease treated consecutively with imiglucerase and miglustat. Calcified tissue international, 2008, Volume: 83, Issue:1 Topics: 1-Deoxynojirimycin; Absorptiometry, Photon; Adult; Aged; Aged, 80 and over; Bone and Bones; Bone Density; Bone Remodeling; Enzyme Inhibitors; Female; Gaucher Disease; Glucosylceramidase; Humans; Magnetic Resonance Imaging; Male; Middle Aged	2008
Glycosphingolipid depletion in PC12 cells using iminosugars protects neuronal membranes from anti-ganglioside antibody mediated injury. Journal of neuroimmunology, 2008, Oct-15, Volume: 203, Issue:1 Topics: 1-Deoxynojirimycin; Animals; Antibodies; Cell Membrane; Complement System Proteins; Enzyme Inhibitors; Gangliosides; Glycosphingolipids; Imino Sugars; Neuritis, Autoimmune, Experimental; Neurons; Neuroprotective Agents; PC12 Cells; Rats	2008
N-Butyldeoxynojirimycin is a broadly effective anti-HIV therapy significantly enhanced by targeted liposome delivery. AIDS (London, England), 2008, Oct-01, Volume: 22, Issue:15 Topics: 1-Deoxynojirimycin; Anti-HIV Agents; Cells, Cultured; Dose-Response Relationship, Drug; Drug Evaluation, Preclinical; Drug Resistance, Viral; HIV Infections; HIV-1; HIV-2; Humans; Leukocytes, Mononuclear; Liposomes; Virulence; Virus Replication	2008
Anti-inflammatory effect of miglustat in bronchial epithelial cells. Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2008, Volume: 7, Issue:6 Topics: 1-Deoxynojirimycin; Bronchi; Cell Culture Techniques; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Enzyme Inhibitors; Epithelial Cells; Humans; Inflammation Mediators; Protein Transport; Respiratory Mucosa	2008
Treatment of a child diagnosed with Niemann-Pick disease type C with miglustat: a case report in Brazil. Journal of inherited metabolic disease, 2008, Volume: 31 Suppl 2 Topics: 1-Deoxynojirimycin; Brazil; Child; Child Behavior; Child Development; Disability Evaluation; Enzyme Inhibitors; Female; Glucosyltransferases; Humans; Magnetic Resonance Imaging; Niemann-Pick Disease, Type C; Recovery of Function; Severity of Illness Index; Time Factors; Treatment Outcome	2008
24 month-treatment with miglustat of three patients with Niemann-Pick disease type C: follow up using brain spectroscopy. Molecular genetics and metabolism, 2009, Volume: 96, Issue:2 Topics: 1-Deoxynojirimycin; Adult; Brain; Choline; Cohort Studies; Creatine; Female; Humans; Magnetic Resonance Imaging; Magnetic Resonance Spectroscopy; Male; Niemann-Pick Disease, Type C; Treatment Outcome	2009
Rapid quantification of miglustat in human plasma and cerebrospinal fluid by liquid chromatography coupled with tandem mass spectrometry. Journal of chromatography. B, Analytical technologies in the biomedical and life sciences, 2009, Jan-15, Volume: 877, Issue:3 Topics: 1-Deoxynojirimycin; Calibration; Child; Chromatography, Liquid; Drug Stability; Enzyme Inhibitors; Humans; Least-Squares Analysis; Reference Standards; Reproducibility of Results; Sensitivity and Specificity; Tandem Mass Spectrometry	2009
A cystic fibrosis respiratory epithelial cell chronically treated by miglustat acquires a non-cystic fibrosis-like phenotype. American journal of respiratory cell and molecular biology, 2009, Volume: 41, Issue:2 Topics: 1-Deoxynojirimycin; Animals; Calcium; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Enzyme Inhibitors; Epithelial Cells; Epithelial Sodium Channels; Homeostasis; Humans; Mice; Patch-Clamp Techniques; Phenotype; Respiratory Mucosa	2009
The pharmacological chaperone N-butyldeoxynojirimycin enhances enzyme replacement therapy in Pompe disease fibroblasts. Molecular therapy : the journal of the American Society of Gene Therapy, 2009, Volume: 17, Issue:6 Topics: 1-Deoxynojirimycin; alpha-Galactosidase; Animals; Biological Transport; Blotting, Western; Cell Line; Drug Stability; Enzyme Inhibitors; Fibroblasts; Glycogen Storage Disease Type II; Humans; Lysosomes; Mice; Microscopy, Confocal	2009
Airway delivery of low-dose miglustat normalizes nasal potential difference in F508del cystic fibrosis mice. American journal of respiratory and critical care medicine, 2009, Jun-01, Volume: 179, Issue:11 Topics: 1-Deoxynojirimycin; Administration, Intranasal; Animals; Chloride Channels; Cystic Fibrosis; Enzyme Inhibitors; Mice; Mice, Knockout; Sodium Channels	2009
Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme. Pharmacoepidemiology and drug safety, 2009, Volume: 18, Issue:9 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Child; Child, Preschool; Enzyme Inhibitors; Europe; Female; Gaucher Disease; Humans; Male; Middle Aged; Nervous System Diseases; Retrospective Studies; Severity of Illness Index; Young Adult	2009
Miglustat in patients with Niemann-Pick disease Type C (NP-C): a multicenter observational retrospective cohort study. Molecular genetics and metabolism, 2009, Volume: 98, Issue:3 Topics: 1-Deoxynojirimycin; Adolescent; Child; Cohort Studies; Enzyme Inhibitors; Female; Humans; Male; Niemann-Pick Disease, Type C; Retrospective Studies; Treatment Outcome	2009
Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression. PloS one, 2009, Sep-11, Volume: 4, Issue:9 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; beta-Cyclodextrins; Cholesterol; Cyclodextrins; Disease Models, Animal; Disease Progression; Drug Synergism; Enzyme Inhibitors; Glycosphingolipids; Mice; Mice, Transgenic; Neurons; Niemann-Pick Disease, Type C; Pregnanolone; Treatment Outcome	2009
Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease. Blood cells, molecules & diseases, 2010, Jan-15, Volume: 44, Issue:1 Topics: 1-Deoxynojirimycin; Africa, Northern; Compassionate Use Trials; Drug Contamination; Drugs, Investigational; Enzyme Inhibitors; Enzyme Replacement Therapy; Equipment Contamination; Europe; Gaucher Disease; Glucosylceramidase; Guidelines as Topic; Health Care Rationing; Health Priorities; Humans; International Cooperation; Middle East; Recombinant Proteins; Vesivirus	2010
Substrate deprivation therapy in juvenile Sandhoff disease. Journal of inherited metabolic disease, 2009, Volume: 32 Suppl 1 Topics: 1-Deoxynojirimycin; Adolescent; Child, Preschool; Disease Progression; Enzyme Inhibitors; Glucosyltransferases; Hexosaminidase B; Humans; Male; Mutation; Sandhoff Disease	2009
Miglustat therapy in juvenile Sandhoff disease. Journal of inherited metabolic disease, 2009, Volume: 32 Suppl 1 Topics: 1-Deoxynojirimycin; Adolescent; beta-Hexosaminidase beta Chain; Cerebellum; Child, Preschool; Depression; Disease Progression; Electroconvulsive Therapy; Enzyme Inhibitors; Glucosyltransferases; Humans; Male; Mutation; Sandhoff Disease	2009
Eye movement and diffusion tensor imaging analysis of treatment effects in a Niemann-Pick Type C patient. Molecular genetics and metabolism, 2010, Volume: 99, Issue:3 Topics: 1-Deoxynojirimycin; Adult; Anisotropy; Diffusion Tensor Imaging; Enzyme Inhibitors; Eye Movements; Humans; Male; Niemann-Pick Disease, Type C; Treatment Outcome; Young Adult	2010
Clinical experience with miglustat therapy in pediatric patients with Niemann-Pick disease type C: a case series. Molecular genetics and metabolism, 2010, Volume: 99, Issue:4 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Age Factors; Child; Child, Preschool; Cognition; Female; Humans; Male; Nervous System Diseases; Niemann-Pick Disease, Type C	2010
Treatment of cataplexy in Niemann-Pick disease type C with the use of miglustat. European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society, 2011, Volume: 15, Issue:1 Topics: 1-Deoxynojirimycin; Cataplexy; Child; Enzyme Inhibitors; Humans; Male; Niemann-Pick Disease, Type C	2011
[Treatment of bone involvement in Gaucher diseases with imiglucerase combined with miglustat]. Presse medicale (Paris, France : 1983), 2009, Volume: 38 Suppl 2 Topics: 1-Deoxynojirimycin; Adult; Bone Diseases; Drug Therapy, Combination; Enzyme Inhibitors; Female; Gaucher Disease; Glucosylceramidase; Humans	2009
C terminus of nucleotide binding domain 1 contains critical features for cystic fibrosis transmembrane conductance regulator trafficking and activation. The Journal of biological chemistry, 2010, Jul-16, Volume: 285, Issue:29 Topics: 1-Deoxynojirimycin; Blotting, Western; Cell Line; Colforsin; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Iodides; Ion Channel Gating; Models, Molecular; Mutant Proteins; Protein Structure, Secondary; Protein Structure, Tertiary; Protein Transport; Quinolizines; Structure-Activity Relationship	2010
How adhesion/growth-regulatory galectins-1 and -3 attain cell specificity: case study defining their target on neuroblastoma cells (SK-N-MC) and marked affinity regulation by affecting microdomain organization of the membrane. IUBMB life, 2010, Volume: 62, Issue:8 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Anti-Bacterial Agents; beta-Cyclodextrins; Cell Adhesion; Cell Membrane; Enzyme Inhibitors; Excipients; Filipin; G(M1) Ganglioside; Galectin 1; Galectin 3; Glycoside Hydrolase Inhibitors; Humans; Membrane Microdomains; Morpholines; Neuroblastoma; Tumor Cells, Cultured	2010
Substrate reduction therapy with miglustat in chronic GM2 gangliosidosis type Sandhoff: results of a 3-year follow-up. Journal of inherited metabolic disease, 2010, Volume: 33 Suppl 3 Topics: 1-Deoxynojirimycin; Disease Progression; Enzyme Inhibitors; Glucosyltransferases; Humans; Magnetic Resonance Imaging; Male; Middle Aged; Muscle Weakness; Muscular Atrophy, Spinal; Neurologic Examination; Predictive Value of Tests; Sandhoff Disease; Time Factors; Treatment Outcome	2010
Dietary modifications in patients receiving miglustat. Journal of inherited metabolic disease, 2010, Volume: 33 Suppl 3 Topics: 1-Deoxynojirimycin; Adolescent; Adolescent Development; Adult; Age Factors; Child; Child Development; Diet, Carbohydrate-Restricted; England; Enzyme Inhibitors; Gastrointestinal Diseases; Glucosyltransferases; Humans; Lactose; Lysosomal Storage Diseases, Nervous System; Retrospective Studies; Time Factors; Treatment Outcome; Weight Loss; Young Adult	2010
Misdiagnosis of Niemann-Pick disease type C as Gaucher disease. Journal of inherited metabolic disease, 2010, Volume: 33 Suppl 3 Topics: 1-Deoxynojirimycin; Acid Phosphatase; Biomarkers; Carrier Proteins; Cells, Cultured; Cholesterol Esters; Diagnostic Errors; DNA Mutational Analysis; Enzyme Inhibitors; Enzyme Replacement Therapy; Esterification; Female; Gaucher Disease; Genetic Predisposition to Disease; Glucosylceramidase; Glucosyltransferases; Hepatomegaly; Heterozygote; Hexosaminidases; Humans; Infant; Intracellular Signaling Peptides and Proteins; Isoenzymes; Membrane Glycoproteins; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Phenotype; Predictive Value of Tests; Splenomegaly; Tartrate-Resistant Acid Phosphatase; Unnecessary Procedures	2010
[Niemann Pick type C disease]. Kinderkrankenschwester : Organ der Sektion Kinderkrankenpflege, 2010, Volume: 29, Issue:9 Topics: 1-Deoxynojirimycin; Brain; Child; Child, Preschool; Chromosome Aberrations; Combined Modality Therapy; Developmental Disabilities; Diagnosis, Differential; Disability Evaluation; Disease Progression; Enzyme Inhibitors; Female; Genes, Recessive; Genetic Testing; Humans; Infant, Newborn; Magnetic Resonance Imaging; Niemann-Pick Disease, Type C; Parenting	2010
Gamma-secretase-dependent amyloid-beta is increased in Niemann-Pick type C: a cross-sectional study. Neurology, 2011, Jan-25, Volume: 76, Issue:4 Topics: 1-Deoxynojirimycin; Adolescent; Amyloid beta-Peptides; Amyloid beta-Protein Precursor; Amyloid Precursor Protein Secretases; Apolipoproteins E; Axons; Biomarkers; Child; Child, Preschool; Cross-Sectional Studies; Female; Genotype; Glucosyltransferases; Humans; Infant; Male; Niemann-Pick Diseases; Peptide Fragments; Phosphoproteins; tau Proteins; Young Adult	2011
White and gray matter alterations in adults with Niemann-Pick disease type C: a cross-sectional study. Neurology, 2011, Jan-11, Volume: 76, Issue:2 Topics: 1-Deoxynojirimycin; Adult; Brain; Case-Control Studies; Cross-Sectional Studies; Diffusion Tensor Imaging; Enzyme Inhibitors; Female; Follow-Up Studies; Humans; Male; Niemann-Pick Disease, Type C; Treatment Outcome	2011
The videofluoroscopic swallowing study shows a sustained improvement of dysphagia in children with Niemann-Pick disease type C after therapy with miglustat. American journal of medical genetics. Part A, 2011, Volume: 155A, Issue:3 Topics: 1-Deoxynojirimycin; Child; Deglutition; Deglutition Disorders; Demography; Enzyme Inhibitors; Female; Fluoroscopy; Humans; Infant; Male; Niemann-Pick Disease, Type C; Video Recording	2011
Modulators of sphingolipid metabolism reduce lung inflammation. American journal of respiratory cell and molecular biology, 2011, Volume: 45, Issue:4 Topics: 1-Deoxynojirimycin; Amitriptyline; Animals; Anti-Inflammatory Agents; Cell Line; Ceramides; Disease Models, Animal; Epithelial Cells; Gene Expression Regulation; Host-Pathogen Interactions; Humans; Immunity, Innate; Inflammation Mediators; Interleukin-8; Lipopolysaccharides; Mice; Mice, Inbred C57BL; Neutrophil Infiltration; Pneumonia; Pseudomonas aeruginosa; Respiratory Mucosa	2011
Glucosylceramide synthase inhibitor PDMP sensitizes chronic myeloid leukemia T315I mutant to Bcr-Abl inhibitor and cooperatively induces glycogen synthase kinase-3-regulated apoptosis. FASEB journal : official publication of the Federation of American Societies for Experimental Biology, 2011, Volume: 25, Issue:10 Topics: 1-Deoxynojirimycin; Animals; Apoptosis; Cell Line, Tumor; Ceramides; Dose-Response Relationship, Drug; Drug Resistance, Neoplasm; Enzyme Inhibitors; Female; Genes, abl; Glucosyltransferases; Glycogen Synthase Kinase 3; Humans; Immunoglobulin G; Leukemia, Myelogenous, Chronic, BCR-ABL Positive; Melphalan; Mice; Mice, SCID; Morpholines; Mutation; Neoplasms, Experimental; Pyrimidines; Transplantation, Heterologous	2011
Gaucher disease due to saposin C deficiency, previously described as non-neuronopathic form--no positive effects after 2-years of miglustat therapy. Molecular genetics and metabolism, 2011, Volume: 104, Issue:4 Topics: 1-Deoxynojirimycin; Adult; Diagnostic Errors; Enzyme Inhibitors; Female; Gaucher Disease; Hepatomegaly; Humans; Male; Saposins; Splenomegaly; Treatment Failure	2011
Endoplasmic reticulum stress induces autophagy through activation of p38 MAPK in fibroblasts from Pompe disease patients carrying c.546G>T mutation. Molecular genetics and metabolism, 2011, Volume: 104, Issue:4 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Autophagy; Cells, Cultured; DNA Mutational Analysis; Endoplasmic Reticulum Stress; Enzyme Activation; Enzyme Activators; Fibroblasts; Glycogen Storage Disease Type II; MAP Kinase Signaling System; p38 Mitogen-Activated Protein Kinases; Phosphoproteins; Phosphorylation; Point Mutation; Protein Folding; Protein Isoforms; Unfolded Protein Response	2011
Extended remission of B-cell lymphoma with monoclonal gammopathy in a patient with type 1 Gaucher disease treated with enzyme replacement therapy. Blood cells, molecules & diseases, 2012, Jan-15, Volume: 48, Issue:1 Topics: 1-Deoxynojirimycin; Enzyme Replacement Therapy; Female; Gaucher Disease; Glucosylceramidase; Humans; Lymphoma, B-Cell; Middle Aged; Monoclonal Gammopathy of Undetermined Significance; Splenectomy	2012
Fusion failure of dense-cored proacrosomal vesicles in an inducible mouse model of male infertility. Cell and tissue research, 2011, Volume: 346, Issue:1 Topics: 1-Deoxynojirimycin; Acrosome; Animals; Asthenozoospermia; Cell Nucleus; Disease Models, Animal; Enzyme Inhibitors; Golgi Apparatus; Male; Membrane Fusion; Mice; Secretory Vesicles; Spermatids; Spermatogenesis; Ultrasonography	2011
Therapeutic strategies for Gaucher disease: miglustat (NB-DNJ) as a pharmacological chaperone for glucocerebrosidase and the different thermostability of velaglucerase alfa and imiglucerase. Molecular pharmaceutics, 2011, Dec-05, Volume: 8, Issue:6 Topics: 1-Deoxynojirimycin; Calorimetry, Differential Scanning; Drug Stability; Enzyme Stability; Gaucher Disease; Glucosylceramidase; Humans; Hydrogen-Ion Concentration; Models, Biological; Molecular Chaperones; Temperature	2011
[Report of attendance at the Parseghian Scientific Conference for Niemann-Pick Type C (NP-C) Research, South Bend, Indiana: in search of new therapeutic possibilities for a very rare illness]. Kinderkrankenschwester : Organ der Sektion Kinderkrankenpflege, 2011, Volume: 30, Issue:10 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Adolescent; Animals; beta-Cyclodextrins; Child; Disease Models, Animal; Diseases in Twins; Drugs, Investigational; Histone Deacetylases; Humans; Infusions, Intravenous; Injections, Spinal; Mice; Niemann-Pick Disease, Type C; Research; United States; United States Food and Drug Administration	2011
Validated LC-MS/MS method for the quantitative determination of the glucosylceramide synthase inhibitor miglustat in mouse plasma and human plasma and its application to a pharmacokinetic study. Journal of pharmaceutical and biomedical analysis, 2012, Feb-05, Volume: 59 Topics: 1-Deoxynojirimycin; Animals; Calibration; Chromatography, High Pressure Liquid; Drug Stability; Enzyme Inhibitors; Glucosyltransferases; Humans; Limit of Detection; Mice; Reference Standards; Reproducibility of Results; Species Specificity; Tandem Mass Spectrometry	2012
Corneal alterations during combined therapy with cyclodextrin/allopregnanolone and miglustat in a knock-out mouse model of NPC1 disease. PloS one, 2011, Volume: 6, Issue:12 Topics: 1-Deoxynojirimycin; Anesthetics; Animals; Chromatography, High Pressure Liquid; Chromatography, Liquid; Cornea; Cyclodextrins; Disease Progression; Drug Therapy, Combination; Enzyme Inhibitors; Genotype; Intracellular Signaling Peptides and Proteins; Lysosomes; Mass Spectrometry; Mice; Mice, Knockout; Microscopy, Confocal; Models, Biological; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Pregnanolone; Proteins	2011
[Guidelines for type 1 Gaucher's disease]. Medicina clinica, 2011, Volume: 137 Suppl 1 Topics: 1-Deoxynojirimycin; Algorithms; Enzyme Replacement Therapy; Gaucher Disease; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Humans	2011
Substrate reduction therapy with miglustat for type 1 Gaucher disease: a retrospective analysis from a single institution. Upsala journal of medical sciences, 2012, Volume: 117, Issue:1 Topics: 1-Deoxynojirimycin; Adult; Aged; Aged, 80 and over; Enzyme Inhibitors; Female; Gaucher Disease; Humans; Male; Middle Aged; Retrospective Studies	2012
Gaucher disease: hematologic and oncologic implications. Clinical advances in hematology & oncology : H&O, 2011, Volume: 9, Issue:10 Topics: 1-Deoxynojirimycin; Carcinoma, Hepatocellular; Enzyme Inhibitors; Enzyme Replacement Therapy; Gaucher Disease; Glucosylceramidase; Glucosyltransferases; Humans; Liver; Multiple Myeloma; Risk Factors; Spleen; Splenectomy	2011
Long-term efficacy of miglustat in paediatric patients with Niemann-Pick disease type C. Journal of inherited metabolic disease, 2013, Volume: 36, Issue:1 Topics: 1-Deoxynojirimycin; Child; Child, Preschool; Cognition; Deglutition; Disease Progression; Drug Administration Schedule; Female; Humans; Male; Nervous System Diseases; Niemann-Pick Disease, Type C; Time; Treatment Outcome	2013
Miglustat improves purkinje cell survival and alters microglial phenotype in feline Niemann-Pick disease type C. Journal of neuropathology and experimental neurology, 2012, Volume: 71, Issue:5 Topics: 1-Deoxynojirimycin; Animals; Antigens, CD; Area Under Curve; Brain; Case-Control Studies; Cat Diseases; Cats; Cell Survival; Cholesterol; Enzyme Inhibitors; Follow-Up Studies; Gangliosidoses, GM2; Microglia; Niemann-Pick Disease, Type C; Phagocytosis; Phenotype; Postmortem Changes; Purkinje Cells; Reactive Oxygen Species; Sphingomyelins; Sphingosine; Time Factors	2012
Metabolic liver disease. Clinics and research in hepatology and gastroenterology, 2012, Volume: 36, Issue:3 Topics: 1-Deoxynojirimycin; Cyclohexanones; Diet; Enzyme Inhibitors; Enzyme Replacement Therapy; Hepatocytes; Hepatomegaly; Humans; Hydrops Fetalis; Infant; Infant, Newborn; Jaundice, Neonatal; Liver Diseases; Liver Transplantation; Metabolism, Inborn Errors; Nitrobenzoates; Splenomegaly	2012
Early miglustat therapy in infantile Niemann-Pick disease type C. Pediatric neurology, 2012, Volume: 47, Issue:1 Topics: 1-Deoxynojirimycin; Carrier Proteins; Child; DNA Mutational Analysis; Enzyme Inhibitors; Female; Humans; Intracellular Signaling Peptides and Proteins; Longitudinal Studies; Membrane Glycoproteins; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C	2012
Initiation and discontinuation of substrate inhibitor treatment in patients with Niemann-Pick type C disease. Gene, 2012, Sep-10, Volume: 506, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Carrier Proteins; Child; Child, Preschool; Developmental Disabilities; Disability Evaluation; Disease Progression; Enzyme Inhibitors; Female; Glucosyltransferases; Humans; Infant; Infant, Newborn; Intracellular Signaling Peptides and Proteins; Language Development Disorders; Male; Membrane Glycoproteins; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C	2012
Oxidative stress in Niemann-Pick type C patients: a protective role of N-butyl-deoxynojirimycin therapy. International journal of developmental neuroscience : the official journal of the International Society for Developmental Neuroscience, 2012, Volume: 30, Issue:6 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Antioxidants; Catalase; Child; Enzyme Inhibitors; Erythrocytes; Female; Fibroblasts; Glutathione; Glutathione Peroxidase; Humans; Infant; Longitudinal Studies; Male; Niemann-Pick Disease, Type C; Oxidative Stress; Plasma; Protein Carbonylation; Statistics, Nonparametric; Superoxide Dismutase; Thiobarbituric Acid Reactive Substances; Young Adult	2012
Attenuation of acetic acid-induced gastric ulcer formation in rats by glucosylceramide synthase inhibitors. Digestive diseases and sciences, 2013, Volume: 58, Issue:2 Topics: 1-Deoxynojirimycin; Acetic Acid; Animals; Apoptosis; Disease Models, Animal; Enzyme Inhibitors; Fumonisins; G(M3) Ganglioside; Gastric Mucosa; Glucosylceramides; Glucosyltransferases; Humans; Male; Morpholines; Rats; Rats, Sprague-Dawley; Sphingolipids; Stomach Ulcer	2013
Miglustat-induced intestinal carbohydrate malabsorption is due to the inhibition of α-glucosidases, but not β-galactosidases. Journal of inherited metabolic disease, 2012, Volume: 35, Issue:6 Topics: 1-Deoxynojirimycin; beta-Galactosidase; Dietary Carbohydrates; Enzyme Inhibitors; Gaucher Disease; Glycoside Hydrolase Inhibitors; Humans; Intestinal Mucosa; Kinetics; Malabsorption Syndromes; Microvilli; Niemann-Pick Disease, Type C	2012
Effects of miglustat on stabilization of neurological disorder in niemann-pick disease type C: Iranian pediatric case series. Journal of child neurology, 2013, Volume: 28, Issue:12 Topics: 1-Deoxynojirimycin; Adolescent; Child; Child, Preschool; Enzyme Inhibitors; Female; Humans; Infant; Iran; Male; Nervous System Diseases; Niemann-Pick Disease, Type C; Retrospective Studies; Treatment Outcome; Young Adult	2013
Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study. Orphanet journal of rare diseases, 2012, Dec-27, Volume: 7 Topics: 1-Deoxynojirimycin; Adult; Enzyme Inhibitors; Enzyme Therapy; Female; Gaucher Disease; Humans; Male; Middle Aged; Prospective Studies; Treatment Outcome	2012
Disease and patient characteristics in NP-C patients: findings from an international disease registry. Orphanet journal of rare diseases, 2013, Jan-16, Volume: 8 Topics: 1-Deoxynojirimycin; Adolescent; Age of Onset; Child; Child, Preschool; Disabled Persons; Enzyme Inhibitors; Female; Humans; Infant; Male; Mutation; Niemann-Pick Disease, Type C; Prospective Studies; Registries	2013
Glycoprotein misfolding in the endoplasmic reticulum: identification of released oligosaccharides reveals a second ER-associated degradation pathway for Golgi-retrieved proteins. Cellular and molecular life sciences : CMLS, 2013, Volume: 70, Issue:15 Topics: 1-Deoxynojirimycin; Animals; Blotting, Western; Cattle; Cell Line; CHO Cells; Chromatography, High Pressure Liquid; Cricetinae; Cricetulus; Digitonin; Endoplasmic Reticulum; Fluorescence; Glycoproteins; Glycoside Hydrolase Inhibitors; Golgi Apparatus; Oligosaccharides; Protein Folding; Proteolysis; Spectrometry, Mass, Matrix-Assisted Laser Desorption-Ionization	2013
Human and mouse neuroinflammation markers in Niemann-Pick disease, type C1. Journal of inherited metabolic disease, 2014, Volume: 37, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Alleles; Animals; Biomarkers; Brain; Cerebellum; Cerebral Cortex; Chemokine CCL3; Chemokine CXCL5; Child; Child, Preschool; Complement C3; Disease Models, Animal; Female; Gene Expression Regulation; Humans; Infant; Infant, Newborn; Inflammation; Interleukins; Male; Mice; Mice, Inbred BALB C; Mice, Transgenic; Niemann-Pick Disease, Type C; Young Adult	2014
Miglustat therapy in type 1 Gaucher disease: clinical and safety outcomes in a multicenter retrospective cohort study. Blood cells, molecules & diseases, 2013, Volume: 51, Issue:2 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Child; Child, Preschool; Female; Gaucher Disease; Glycoside Hydrolase Inhibitors; Hemoglobins; Humans; Infant; Infant, Newborn; Male; Middle Aged; Retrospective Studies; Treatment Outcome; Young Adult	2013
C-branched iminosugars: α-glucosidase inhibition by enantiomers of isoDMDP, isoDGDP, and isoDAB-L-isoDMDP compared to miglitol and miglustat. The Journal of organic chemistry, 2013, Aug-02, Volume: 78, Issue:15 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Angiogenesis Inhibitors; Biological Products; Dose-Response Relationship, Drug; Enzyme Inhibitors; Glycoside Hydrolase Inhibitors; Imino Sugars; Molecular Conformation; Stereoisomerism; Structure-Activity Relationship	2013
Supersite of immune vulnerability on the glycosylated face of HIV-1 envelope glycoprotein gp120. Nature structural & molecular biology, 2013, Volume: 20, Issue:7 Topics: 1-Deoxynojirimycin; Alkaloids; Amino Acid Motifs; Amino Acid Sequence; Antibodies, Neutralizing; Antigen-Antibody Reactions; Binding Sites, Antibody; Biopolymers; Carbohydrate Sequence; CD4 Antigens; Crystallography, X-Ray; env Gene Products, Human Immunodeficiency Virus; Epitopes; Glycosylation; HEK293 Cells; HIV Antibodies; HIV Envelope Protein gp120; Humans; Immunoglobulin Fab Fragments; Microscopy, Electron; Models, Molecular; Molecular Docking Simulation; Molecular Sequence Data; Polysaccharides; Protein Conformation; Protein Processing, Post-Translational; Structure-Activity Relationship	2013
Identifying chemicals with potential therapy of HIV based on protein-protein and protein-chemical interaction network. PloS one, 2013, Volume: 8, Issue:6 Topics: 1-Deoxynojirimycin; Algorithms; Anti-HIV Agents; CCR5 Receptor Antagonists; Computer Simulation; Cyclohexanes; Databases, Chemical; Didanosine; Drug Design; Drug Discovery; HIV Infections; HIV-1; Host-Pathogen Interactions; Humans; Maraviroc; Models, Molecular; Protein Interaction Mapping; Protein Interaction Maps; Receptors, CCR5; Triazoles	2013
Adult form of Niemann-Pick type C with the variant biochemical phenotype on treatment with Miglustat. Parkinsonism & related disorders, 2013, Volume: 19, Issue:10 Topics: 1-Deoxynojirimycin; Activities of Daily Living; Adult; Anti-Bacterial Agents; Carrier Proteins; Disability Evaluation; Enzyme Inhibitors; Filipin; Glycoside Hydrolase Inhibitors; Humans; Intracellular Signaling Peptides and Proteins; Male; Membrane Glycoproteins; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Phenotype; Psychomotor Performance; Saccades	2013
Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling. Journal of lipid research, 2013, Volume: 54, Issue:10 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; beta-Cyclodextrins; Biomarkers; Cats; Chromatography, High Pressure Liquid; Drug Evaluation, Preclinical; Female; Gangliosides; Humans; Male; Mice; Mice, Inbred BALB C; Mice, Knockout; Niemann-Pick Disease, Type C; Organ Specificity; Sphingolipids; Sulfoglycosphingolipids; Tandem Mass Spectrometry	2013
Niemann-Pick type C: a potentially treatable disorder? Practical neurology, 2013, Volume: 13, Issue:6 Topics: 1-Deoxynojirimycin; Adult; Enzyme Inhibitors; Humans; Male; Niemann-Pick Disease, Type C	2013
Combined therapy with cyclodextrin/allopregnanolone and miglustat improves motor but not cognitive functions in Niemann-Pick Type C1 mice. Neuroscience, 2013, Nov-12, Volume: 252 Topics: 1-Deoxynojirimycin; Animals; Cognition; Cyclodextrins; Disease Models, Animal; Drug Therapy, Combination; Mice; Mice, Knockout; Microscopy, Electron, Transmission; Motor Activity; Neuroprotective Agents; Niemann-Pick Disease, Type C; Pregnanolone	2013
Efficacy of miglustat in Niemann-Pick C disease: a single centre experience. Molecular genetics and metabolism, 2013, Volume: 110, Issue:3 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Age of Onset; Child; Child, Preschool; Delayed Diagnosis; Disabled Persons; Enzyme Inhibitors; Female; Glycoside Hydrolase Inhibitors; Humans; Male; Niemann-Pick Disease, Type C; Retrospective Studies; Treatment Outcome; Young Adult	2013
Complete recovery from psychosis upon miglustat treatment in a juvenile Niemann-Pick C patient. European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society, 2014, Volume: 18, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Age of Onset; Atrophy; Cerebrum; Humans; Male; Niemann-Pick Disease, Type C; Psychotic Disorders; Treatment Outcome	2014
Altered transition metal homeostasis in Niemann-Pick disease, type C1. Metallomics : integrated biometal science, 2014, Volume: 6, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Brain; Ceruloplasmin; Enzyme Inhibitors; Female; Gene Deletion; Homeostasis; Humans; Intracellular Signaling Peptides and Proteins; Male; Metals; Mice; Mice, Inbred BALB C; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Proteins; Transition Elements	2014
SERCA2 dysfunction in Darier disease causes endoplasmic reticulum stress and impaired cell-to-cell adhesion strength: rescue by Miglustat. The Journal of investigative dermatology, 2014, Volume: 134, Issue:7 Topics: 1-Deoxynojirimycin; Adherens Junctions; beta Catenin; Cadherins; Calcium; Cell Adhesion; Cells, Cultured; Darier Disease; Desmosomes; Endoplasmic Reticulum Stress; Enzyme Inhibitors; Female; Humans; Keratinocytes; Male; Sarcoplasmic Reticulum Calcium-Transporting ATPases; Thapsigargin	2014
Normalizing glycosphingolipids restores function in CD4+ T cells from lupus patients. The Journal of clinical investigation, 2014, Volume: 124, Issue:2 Topics: 1-Deoxynojirimycin; Adult; Aged; Antigens, CD; B-Lymphocytes; CD4-Positive T-Lymphocytes; Female; Flow Cytometry; G(M1) Ganglioside; Gene Expression Regulation; Glycosphingolipids; Homeostasis; Humans; Lactosylceramides; Leukocytes, Mononuclear; Liver X Receptors; Lupus Erythematosus, Systemic; Lymphocyte Activation; Male; Membrane Microdomains; Middle Aged; Orphan Nuclear Receptors; Signal Transduction; Time Factors; Trihexosylceramides	2014
Relative acidic compartment volume as a lysosomal storage disorder-associated biomarker. The Journal of clinical investigation, 2014, Volume: 124, Issue:3 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; B-Lymphocytes; beta-Cyclodextrins; Biomarkers; Bone Marrow Transplantation; Case-Control Studies; Child; Child, Preschool; Humans; Infant; Intracellular Signaling Peptides and Proteins; Lysosomes; Mice; Mice, Inbred BALB C; Mice, Knockout; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Prospective Studies; Proteins; Severity of Illness Index; Treatment Outcome	2014
Enhanced F508del-CFTR channel activity ameliorates bone pathology in murine cystic fibrosis. The American journal of pathology, 2014, Volume: 184, Issue:4 Topics: 1-Deoxynojirimycin; Animals; Bone and Bones; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Models, Animal; Enzyme Inhibitors; Male; Mice, Inbred CFTR; Mutation; Osteoblasts	2014
Improved neuroprotection using miglustat, curcumin and ibuprofen as a triple combination therapy in Niemann-Pick disease type C1 mice. Neurobiology of disease, 2014, Volume: 67 Topics: 1-Deoxynojirimycin; Animals; Cerebellum; Curcumin; Drug Therapy, Combination; Ibuprofen; Intracellular Signaling Peptides and Proteins; Mice; Mice, Inbred BALB C; Mice, Knockout; Neuroprotective Agents; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Proteins	2014
Long term differential consequences of miglustat therapy on intestinal disaccharidases. Journal of inherited metabolic disease, 2014, Volume: 37, Issue:6 Topics: 1-Deoxynojirimycin; Caco-2 Cells; Disaccharidases; Gaucher Disease; Glycoproteins; Glycoside Hydrolase Inhibitors; Glycosylation; Humans; Intestines; Membrane Microdomains; Niemann-Pick Disease, Type C; Protein Transport	2014
Endoplasmic reticulum calcium, stress, and cell-to-cell adhesion. The Journal of investigative dermatology, 2014, Volume: 134, Issue:7 Topics: 1-Deoxynojirimycin; Cell Adhesion; Darier Disease; Endoplasmic Reticulum Stress; Female; Humans; Male; Sarcoplasmic Reticulum Calcium-Transporting ATPases	2014
Searching for combinations of small-molecule correctors to restore f508del-cystic fibrosis transmembrane conductance regulator function and processing. The Journal of pharmacology and experimental therapeutics, 2014, Volume: 350, Issue:3 Topics: 1-Deoxynojirimycin; Aminopyridines; Benzamides; Benzodioxoles; Cell Survival; Colforsin; Cystic Fibrosis Transmembrane Conductance Regulator; Dose-Response Relationship, Drug; HeLa Cells; Humans; Thiazoles	2014
A chaperone enhances blood α-glucosidase activity in Pompe disease patients treated with enzyme replacement therapy. Molecular therapy : the journal of the American Society of Gene Therapy, 2014, Volume: 22, Issue:11 Topics: 1-Deoxynojirimycin; Adolescent; Adult; alpha-Glucosidases; Animals; Child; Child, Preschool; Disease Models, Animal; Dried Blood Spot Testing; Drug Synergism; Enzyme Replacement Therapy; Enzyme Stability; Female; Glycogen Storage Disease Type II; Glycoside Hydrolase Inhibitors; Humans; Male; Mice; Middle Aged; Young Adult	2014
GBA2-encoded β-glucosidase activity is involved in the inflammatory response to Pseudomonas aeruginosa. PloS one, 2014, Volume: 9, Issue:8 Topics: 1-Deoxynojirimycin; beta-Glucosidase; Bronchi; Cystic Fibrosis; Epithelial Cells; Glucosylceramidase; Glycoside Hydrolase Inhibitors; Humans; Inflammation; Pseudomonas aeruginosa; Pseudomonas Infections	2014
Strain-specific antiviral activity of iminosugars against human influenza A viruses. The Journal of antimicrobial chemotherapy, 2015, Volume: 70, Issue:1 Topics: 1-Deoxynojirimycin; Antiviral Agents; Humans; Influenza A Virus, H1N1 Subtype; Influenza A Virus, H3N2 Subtype; Microbial Sensitivity Tests; Virus Replication	2015
Effects of miglustat treatment in a patient affected by an atypical form of Tangier disease. Orphanet journal of rare diseases, 2014, Sep-18, Volume: 9 Topics: 1-Deoxynojirimycin; ATP Binding Cassette Transporter 1; Female; Humans; Middle Aged; Tangier Disease; Treatment Outcome	2014
Reduced cerebellar neurodegeneration after combined therapy with cyclodextrin/allopregnanolone and miglustat in NPC1: a mouse model of Niemann-Pick type C1 disease. Journal of neuroscience research, 2015, Volume: 93, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Cell Count; Cerebellum; Cyclodextrins; Disease Models, Animal; Disease Progression; Drug Therapy, Combination; Enzyme Inhibitors; Mice; Nerve Degeneration; Neurons; Niemann-Pick Disease, Type C; Pregnanolone	2015
Neurological and cardiac responses after treatment with miglustat and a ketogenic diet in a patient with Sandhoff disease. European journal of medical genetics, 2015, Volume: 58, Issue:3 Topics: 1-Deoxynojirimycin; Child; Child, Preschool; Diet, Ketogenic; Humans; Male; Sandhoff Disease	2015
Stability of refrigerated miglustat after preparation in InOrpha(®) flavored suspending excipient for compounding of oral solutions and suspensions. Drug design, development and therapy, 2015, Volume: 9 Topics: 1-Deoxynojirimycin; Administration, Oral; Chemistry, Pharmaceutical; Cold Temperature; Color; Drug Compounding; Drug Contamination; Drug Stability; Excipients; Flavoring Agents; Hydrogen-Ion Concentration; Pharmaceutical Solutions; Refrigeration; Solubility; Technology, Pharmaceutical; Time Factors	2015
Results from a 9-year Intensive Safety Surveillance Scheme (IS(3) ) in miglustat (Zavesca(®) )-treated patients. Pharmacoepidemiology and drug safety, 2015, Volume: 24, Issue:3 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Female; Follow-Up Studies; Gaucher Disease; Humans; Male; Product Surveillance, Postmarketing; Prospective Studies; Treatment Outcome	2015
Clinical utility of neuronal cells directly converted from fibroblasts of patients for neuropsychiatric disorders: studies of lysosomal storage diseases and channelopathy. Current molecular medicine, 2015, Volume: 15, Issue:2 Topics: 1-Deoxynojirimycin; Action Potentials; Basic Helix-Loop-Helix Transcription Factors; Cell Differentiation; Epilepsies, Myoclonic; Fibroblasts; G(M2) Ganglioside; Gene Expression; Genetic Vectors; Glycoside Hydrolase Inhibitors; Humans; Lentivirus; Nerve Tissue Proteins; Neurons; Plasmids; Primary Cell Culture; Tay-Sachs Disease; Transcription Factors; Transduction, Genetic; Transgenes	2015
Looking for protein stabilizing drugs with thermal shift assay. Drug testing and analysis, 2015, Volume: 7, Issue:9 Topics: 1-Deoxynojirimycin; alpha-Galactosidase; Drug Evaluation, Preclinical; Enzyme Stability; Fabry Disease; Glycogen Storage Disease Type II; High-Throughput Screening Assays; Humans; Temperature	2015
[Niemann-Pick disease--a rare disease in children's hepatology (a clinical case)]. Eksperimental'naia i klinicheskaia gastroenterologiia = Experimental & clinical gastroenterology, 2014, Issue:8 Topics: 1-Deoxynojirimycin; Adolescent; Carrier Proteins; Female; Humans; Intracellular Signaling Peptides and Proteins; Liver; Membrane Glycoproteins; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Treatment Outcome	2014
Glycosphingolipid synthesis inhibition limits osteoclast activation and myeloma bone disease. The Journal of clinical investigation, 2015, Volume: 125, Issue:6 Topics: 1-Deoxynojirimycin; Animals; Cell Line; CSK Tyrosine-Protein Kinase; Female; Glucosyltransferases; Glycoside Hydrolase Inhibitors; Glycosphingolipids; Insulin-Like Growth Factor I; Membrane Microdomains; Mice; Mice, Knockout; Multiple Myeloma; Osteoclasts; Osteolysis; RANK Ligand; src-Family Kinases; TNF Receptor-Associated Factor 6	2015
Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: an observational cohort study. Orphanet journal of rare diseases, 2015, May-28, Volume: 10 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Child; Child, Preschool; Cohort Studies; Disease Progression; Enzyme Inhibitors; Female; Humans; Male; Niemann-Pick Disease, Type C; Prospective Studies; Treatment Outcome; Young Adult	2015
Longitudinal changes in cerebellar and subcortical volumes in adult-onset Niemann-Pick disease type C patients treated with miglustat. Journal of neurology, 2015, Volume: 262, Issue:9 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Caudate Nucleus; Cerebellum; Disease Progression; Enzyme Inhibitors; Female; Humans; Longitudinal Studies; Magnetic Resonance Imaging; Male; Middle Aged; Niemann-Pick Disease, Type C; Organ Size; Putamen; Thalamus; Young Adult	2015
Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series. Journal of medical case reports, 2015, Jun-23, Volume: 9 Topics: 1-Deoxynojirimycin; Aged, 80 and over; Enzyme Inhibitors; Gaucher Disease; Glucosylceramidase; Humans; Male; Treatment Outcome	2015
Glucocerebrosidase deficiency and mitochondrial impairment in experimental Parkinson disease. Journal of the neurological sciences, 2015, Sep-15, Volume: 356, Issue:1-2 Topics: 1-Deoxynojirimycin; 1-Methyl-4-phenyl-1,2,3,6-tetrahydropyridine; Animals; Brain; Cell Count; Cells, Cultured; Disease Models, Animal; Embryo, Mammalian; Gaucher Disease; Glycoside Hydrolase Inhibitors; Histocompatibility Antigens; Inositol; L-Lactate Dehydrogenase; Male; Mesencephalon; Mice; Mice, Inbred C57BL; Mitochondrial Diseases; Neurons; Parkinsonian Disorders; Tyrosine 3-Monooxygenase	2015
Miglustat Does Not Prevent Neurological Involvement in Niemann Pick C Disease. Pediatric neurology, 2015, Volume: 53, Issue:4 Topics: 1-Deoxynojirimycin; Central Nervous System Agents; Child; Deglutition Disorders; Female; Humans; Male; Motor Activity; Niemann-Pick Disease, Type C; Saccades; Treatment Outcome	2015
Prevention of renal damage caused by Shiga toxin type 2: Action of Miglustat on human endothelial and epithelial cells. Toxicon : official journal of the International Society on Toxinology, 2015, Volume: 105 Topics: 1-Deoxynojirimycin; Cells, Cultured; Endothelium; Epithelium; Humans; Kidney; Shiga Toxin	2015
Urinary excretion and metabolism of miglustat and valproate in patients with Niemann-Pick type C1 disease: One- and two-dimensional solution-state (1)H NMR studies. Journal of pharmaceutical and biomedical analysis, 2016, Jan-05, Volume: 117 Topics: 1-Deoxynojirimycin; Animals; Cohort Studies; Enzyme Inhibitors; Humans; Magnetic Resonance Spectroscopy; Male; Niemann-Pick Disease, Type C; Protons; Rats; Rats, Sprague-Dawley; Valproic Acid	2016
Longitudinal assessment of reflexive and volitional saccades in Niemann-Pick Type C disease during treatment with miglustat. Orphanet journal of rare diseases, 2015, Dec-21, Volume: 10 Topics: 1-Deoxynojirimycin; Adult; Enzyme Inhibitors; Humans; Longitudinal Studies; Niemann-Pick Disease, Type C; Saccades; Young Adult	2015
Miglustat Reverts the Impairment of Synaptic Plasticity in a Mouse Model of NPC Disease. Neural plasticity, 2016, Volume: 2016 Topics: 1-Deoxynojirimycin; Animals; Disease Models, Animal; Enzyme Inhibitors; Extracellular Signal-Regulated MAP Kinases; Hippocampus; Mice; Neuronal Plasticity; Niemann-Pick Disease, Type C; Phosphorylation; Synapses	2016
Characterization of the Zebrafish Homolog of β-Glucosidase 2: A Target of the Drug Miglustat. Zebrafish, 2016, Volume: 13, Issue:3 Topics: 1-Deoxynojirimycin; Amino Acid Sequence; Animals; beta-Glucosidase; Cloning, Molecular; Gene Expression Regulation, Enzymologic; Glycoside Hydrolase Inhibitors; Zebrafish; Zebrafish Proteins	2016
Normalisation of brain spectroscopy findings in Niemann-Pick disease type C patients treated with miglustat. Journal of neurology, 2016, Volume: 263, Issue:5 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aspartic Acid; Brain; Choline; Disability Evaluation; Disease Progression; Enzyme Inhibitors; Female; Follow-Up Studies; Humans; Linear Models; Longitudinal Studies; Male; Niemann-Pick Disease, Type C; Patient Dropouts; Treatment Outcome; Young Adult	2016
Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients. Orphanet journal of rare diseases, 2016, Mar-24, Volume: 11 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Enzyme Replacement Therapy; Female; Gaucher Disease; Glucosylceramidase; Glucosylceramides; Humans; Male; Pyrrolidines	2016
N-butyldeoxynojirimycin treatment restores the innate fear response and improves learning in mucopolysaccharidosis IIIA mice. Molecular genetics and metabolism, 2016, Volume: 118, Issue:2 Topics: 1-Deoxynojirimycin; Analysis of Variance; Animals; Brain; Chemokine CCL3; Disease Models, Animal; Fear; Gangliosides; Glucosyltransferases; Glycoside Hydrolase Inhibitors; Interleukin-1beta; Maze Learning; Mice; Mucopolysaccharidosis III	2016
A neonate with abdominal distension and failure to thrive. Archives of disease in childhood. Education and practice edition, 2017, Volume: 102, Issue:3 Topics: 1-Deoxynojirimycin; Abdomen; Failure to Thrive; Glycoside Hydrolase Inhibitors; Humans; Hydrops Fetalis; Infant, Newborn; Lysosomal Storage Diseases; Male; Treatment Outcome	2017
Selective screening of Niemann-Pick type C Brazilian patients by cholestane-3β,5α,6β-triol and chitotriosidase measurements followed by filipin staining and NPC1/NPC2 gene analysis. Clinica chimica acta; international journal of clinical chemistry, 2016, Aug-01, Volume: 459 Topics: 1-Deoxynojirimycin; Brazil; Carrier Proteins; Cholestanols; Filipin; Glycoproteins; Hexosaminidases; Humans; Intracellular Signaling Peptides and Proteins; Membrane Glycoproteins; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Staining and Labeling; Vesicular Transport Proteins	2016
Visceral symptoms as a key diagnostic sign for the early infantile form of Niemann-Pick disease type C in a Russian patient: a case report. Journal of medical case reports, 2016, Jun-01, Volume: 10, Issue:1 Topics: 1-Deoxynojirimycin; Child, Preschool; Cholestasis, Intrahepatic; Diagnostic Errors; Galactosemias; Genetic Testing; Glycoside Hydrolase Inhibitors; Hepatomegaly; Humans; Infant; Infant, Newborn; Jaundice, Neonatal; Liver; Male; Niemann-Pick Disease, Type C; Russia; Splenomegaly	2016
Anti-amyloidogenic effects of glycosphingolipid synthesis inhibitors occur independently of ganglioside alterations. Molecular and cellular neurosciences, 2016, Volume: 75 Topics: 1-Deoxynojirimycin; Amyloid beta-Peptides; Cell Line, Tumor; Enzyme Inhibitors; Gangliosides; Humans; Morpholines; Neurons	2016
Impact of miglustat on evolution of atypical presentation of late-infantile-onset Niemann-Pick disease type C with early cognitive impairment, behavioral dysfunction, epilepsy, ophthalmoplegia, and cerebellar involvement: a case report. Journal of medical case reports, 2016, Sep-06, Volume: 10, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Carrier Proteins; Cerebellum; Cognition Disorders; Enzyme Inhibitors; Epilepsy; Humans; Intracellular Signaling Peptides and Proteins; Magnetic Resonance Imaging; Male; Membrane Glycoproteins; Mental Disorders; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Ophthalmoplegia; Treatment Outcome	2016
Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report. Journal of medical case reports, 2016, Nov-08, Volume: 10, Issue:1 Topics: 1-Deoxynojirimycin; Adult; Drug Administration Schedule; Drug Eruptions; Enzyme Inhibitors; Enzyme Replacement Therapy; Gaucher Disease; Humans; Male; Quality of Life; Spleen; Treatment Outcome	2016
Pharmacologic Treatment Assigned for Niemann Pick Type C1 Disease Partly Changes Behavioral Traits in Wild-Type Mice. International journal of molecular sciences, 2016, Nov-09, Volume: 17, Issue:11 Topics: 1-Deoxynojirimycin; Animals; Behavior, Animal; Body Weight; Brain; Cognition; Cyclodextrins; Disease Models, Animal; Drug Administration Schedule; Drug Combinations; Hypolipidemic Agents; Male; Maze Learning; Mice; Mice, Inbred BALB C; Niemann-Pick Disease, Type C; Organ Size; Pregnanolone	2016
Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project. Blood cells, molecules & diseases, 2018, Volume: 68 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Female; Follow-Up Studies; Gaucher Disease; Glycoside Hydrolase Inhibitors; Humans; Liver; Male; Middle Aged; Organ Size; Prospective Studies; Spleen; Young Adult	2018
Assessment of the potential for host-targeted iminosugars UV-4 and UV-5 activity against filovirus infections in vitro and in vivo. Antiviral research, 2017, Volume: 138 Topics: 1-Deoxynojirimycin; Animals; Antiviral Agents; Chlorocebus aethiops; Disease Models, Animal; Dose-Response Relationship, Drug; Ebolavirus; Imino Sugars; Macaca; Marburgvirus; Mice; Models, Animal; Vero Cells	2017
Combination therapy in a patient with chronic neuronopathic Gaucher disease: a case report. Journal of medical case reports, 2017, Jan-20, Volume: 11, Issue:1 Topics: 1-Deoxynojirimycin; Administration, Intravenous; Blood-Brain Barrier; Child; Chronic Disease; Combined Modality Therapy; Enzyme Inhibitors; Enzyme Replacement Therapy; Gaucher Disease; Glucosylceramidase; Hexosaminidases; Humans; Male	2017
Patients with Gaucher type 1: Switching from imiglucerase to miglustat therapy. Blood cells, molecules & diseases, 2018, Volume: 68 Topics: 1-Deoxynojirimycin; Adult; Enzyme Inhibitors; Enzyme Replacement Therapy; Female; Gaucher Disease; Glucosylceramidase; Humans; Recombinant Proteins; Treatment Outcome	2018
Evolution of structural neuroimaging biomarkers in a series of adult patients with Niemann-Pick type C under treatment. Orphanet journal of rare diseases, 2017, 02-02, Volume: 12, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Biomarkers; Brain; Case-Control Studies; Child; Child, Preschool; Glycoside Hydrolase Inhibitors; Humans; Middle Aged; Neuroimaging; Niemann-Pick Disease, Type C; Young Adult	2017
A new framework for evaluating the health impacts of treatment for Gaucher disease type 1. Orphanet journal of rare diseases, 2017, 02-20, Volume: 12, Issue:1 Topics: 1-Deoxynojirimycin; Enzyme Inhibitors; Enzyme Replacement Therapy; Gaucher Disease; Glucosylceramidase; Humans	2017
Increased Regenerative Capacity of the Olfactory Epithelium in Niemann-Pick Disease Type C1. International journal of molecular sciences, 2017, Apr-06, Volume: 18, Issue:4 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; Apoptosis; beta-Cyclodextrins; Cell Proliferation; Disease Models, Animal; Humans; Intracellular Signaling Peptides and Proteins; Mice; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Olfactory Mucosa; Pregnanolone; Proteins	2017
Niemann-Pick type C as a cause of progressive intellectual and neurological deterioration in childhood. Developmental medicine and child neurology, 2017, Volume: 59, Issue:9 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Age of Onset; Brain; Child; Child, Preschool; Delayed Diagnosis; Disease Progression; Epidemiological Monitoring; Female; Follow-Up Studies; Glycoside Hydrolase Inhibitors; Humans; Infant; Intellectual Disability; Male; Niemann-Pick Disease, Type C; Prospective Studies; Time Factors; United Kingdom	2017
The treatment of juvenile/adult GM1-gangliosidosis with Miglustat may reverse disease progression. Metabolic brain disease, 2017, Volume: 32, Issue:5 Topics: 1-Deoxynojirimycin; Adult; Age Factors; beta-Galactosidase; Cognition Disorders; Disease Progression; Female; Follow-Up Studies; Gait; Gangliosidosis, GM1; Genotype; Glycoside Hydrolase Inhibitors; Humans; Language Disorders; Motor Skills; Movement Disorders; Neuropsychological Tests; Treatment Outcome; Walking; Young Adult	2017
Miglustat therapy in a case of early-infantile Niemann-Pick type C. Brain & development, 2017, Volume: 39, Issue:10 Topics: 1-Deoxynojirimycin; Base Sequence; Carrier Proteins; Child, Preschool; Developmental Disabilities; Disease Progression; Glucosyltransferases; Humans; Intracellular Signaling Peptides and Proteins; Japan; Male; Membrane Glycoproteins; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C	2017
N-butyldeoxynojirimycin delays motor deficits, cerebellar microgliosis, and Purkinje cell loss in a mouse model of mucolipidosis type IV. Neurobiology of disease, 2017, Volume: 105 Topics: 1-Deoxynojirimycin; Animals; Antigens, CD; Cell Count; Cerebellum; Disease Models, Animal; Enzyme Inhibitors; Exploratory Behavior; Gliosis; Lipid Metabolism; Mice; Mice, Inbred C57BL; Mice, Transgenic; Movement Disorders; Mucolipidoses; Nerve Tissue Proteins; Psychomotor Performance; Purkinje Cells; Retina; Transient Receptor Potential Channels	2017
NMR analysis reveals significant differences in the plasma metabolic profiles of Niemann Pick C1 patients, heterozygous carriers, and healthy controls. Scientific reports, 2017, 07-24, Volume: 7, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Biomarkers; Case-Control Studies; Child; Child, Preschool; Female; Heterozygote; Humans; Infant; Isoleucine; Male; Metabolomics; Niemann-Pick Disease, Type C; Proton Magnetic Resonance Spectroscopy; Triglycerides; Young Adult	2017
Patient with Niemann-Pick disease type C: over 20 years' follow-up. BMJ case reports, 2017, Aug-22, Volume: 2017 Topics: 1-Deoxynojirimycin; Adult; Diagnosis, Differential; Enzyme Inhibitors; Female; Follow-Up Studies; Humans; Magnetic Resonance Imaging; Niemann-Pick Disease, Type C	2017
Alteration of cortical excitability and its modulation by Miglustat in Niemann-Pick disease type C. Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia, 2018, Volume: 47 Topics: 1-Deoxynojirimycin; Cerebellum; Cognition; Cortical Excitability; Glycoside Hydrolase Inhibitors; Humans; Male; Niemann-Pick Disease, Type C; Transcranial Magnetic Stimulation; Young Adult	2018
N-Butyl-l-deoxynojirimycin (l-NBDNJ): Synthesis of an Allosteric Enhancer of α-Glucosidase Activity for the Treatment of Pompe Disease. Journal of medicinal chemistry, 2017, 12-14, Volume: 60, Issue:23 Topics: 1-Deoxynojirimycin; Allosteric Regulation; alpha-Glucosidases; Cell Line; Enzyme Activation; Enzyme Inhibitors; Fibroblasts; Glycogen Storage Disease Type II; Humans; Lysosomes; Models, Molecular; Stereoisomerism	2017
Molecular and biochemical biomarkers for diagnosis and therapy monitorization of Niemann-Pick type C patients. International journal of developmental neuroscience : the official journal of the International Society for Developmental Neuroscience, 2018, Volume: 66 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Child; Cholestanols; Female; Filipin; Glycoside Hydrolase Inhibitors; Hexosaminidases; Humans; Male; Membrane Glycoproteins; Mutation; Niemann-Pick Disease, Type C; Skin; Young Adult	2018
GBA1 deficiency negatively affects physiological α-synuclein tetramers and related multimers. Proceedings of the National Academy of Sciences of the United States of America, 2018, 01-23, Volume: 115, Issue:4 Topics: 1-Deoxynojirimycin; alpha-Synuclein; Cell Line, Tumor; Dopaminergic Neurons; Glucosylceramidase; Glycosphingolipids; Humans; Parkinson Disease; Protein Multimerization	2018
Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports. Journal of medical case reports, 2018, Jan-27, Volume: 12, Issue:1 Topics: 1-Deoxynojirimycin; Adult; Child, Preschool; Combined Modality Therapy; Drug Therapy, Combination; Enzyme Inhibitors; Enzyme Replacement Therapy; Female; Gaucher Disease; Glucosylceramidase; Hexosaminidases; Humans; Thrombocytopenia	2018
Long-term therapy with miglustat and cognitive decline in the adult form of Niemann-Pick disease type C: a case report. Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology, 2018, Volume: 39, Issue:6 Topics: 1-Deoxynojirimycin; Adult; Cognitive Dysfunction; Enzyme Inhibitors; Female; Humans; Niemann-Pick Disease, Type C; Siblings; Treatment Failure	2018
Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect. Orphanet journal of rare diseases, 2018, 10-01, Volume: 13, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Enzyme Inhibitors; Female; France; Humans; Male; Middle Aged; Niemann-Pick Disease, Type C; Retrospective Studies; Young Adult	2018
Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review. Orphanet journal of rare diseases, 2019, 01-08, Volume: 14, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Child; Child, Preschool; Female; Humans; Linear Models; Male; Niemann-Pick Disease, Type C; Prognosis; Retrospective Studies	2019
Improved efficacy of a next-generation ERT in murine Pompe disease. JCI insight, 2019, 03-07, Volume: 4, Issue:5 Topics: 1-Deoxynojirimycin; alpha-Glucosidases; Animals; Disease Models, Animal; Enzyme Replacement Therapy; Female; Glycogen; Glycogen Storage Disease Type II; Humans; Lysosomes; Male; Mannosephosphates; Mice; Mice, Knockout; Muscle, Skeletal; Rats; Rats, Sprague-Dawley	2019
Niemann-Pick type C: contemporary diagnosis and treatment of a classical disorder. Practical neurology, 2019, Volume: 19, Issue:5 Topics: 1-Deoxynojirimycin; Adult; Female; Humans; Lysosomal Storage Diseases; Neurodegenerative Diseases; Niemann-Pick Disease, Type C; Skin; Treatment Outcome	2019
A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1. Lipids in health and disease, 2019, Jun-28, Volume: 18, Issue:1 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; Cell Separation; Disease Models, Animal; Flow Cytometry; Genotype; Lipid Metabolism; Mice; Niemann-Pick Disease, Type C; Pregnanolone; Spleen	2019
Mechanistic convergence and shared therapeutic targets in Niemann-Pick disease. Journal of inherited metabolic disease, 2020, Volume: 43, Issue:3 Topics: 1-Deoxynojirimycin; Adult; ATP Binding Cassette Transporter 1; Humans; Intracellular Signaling Peptides and Proteins; Middle Aged; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Treatment Outcome	2020
Pre-diagnosing and managing patients with GM1 gangliosidosis and related disorders by the evaluation of GM1 ganglioside content. Scientific reports, 2019, 11-27, Volume: 9, Issue:1 Topics: 1-Deoxynojirimycin; Biomarkers; Cells, Cultured; Disease Progression; Female; Fibroblasts; Flow Cytometry; G(M1) Ganglioside; Gangliosidosis, GM1; Glycoside Hydrolase Inhibitors; Humans; Lymphocytes; Male; Optical Imaging; Phenotype; Severity of Illness Index	2019
Metabolic Control of Astrocyte Pathogenic Activity via cPLA2-MAVS. Cell, 2019, 12-12, Volume: 179, Issue:7 Topics: 1-Deoxynojirimycin; Adaptor Proteins, Signal Transducing; Animals; Astrocytes; Brain; Cells, Cultured; Encephalomyelitis, Autoimmune, Experimental; Female; Hexokinase; Humans; Lactic Acid; Male; Mice; Mice, Inbred C57BL; NF-kappa B; Phospholipases A2, Secretory	2019
The The Journal of biological chemistry, 2020, 01-17, Volume: 295, Issue:3 Topics: 1-Deoxynojirimycin; Animals; Arabidopsis; Arabidopsis Proteins; Biosynthetic Pathways; Glucosylceramidase; Glucosylceramides; Humans; Metabolism; Microtubule-Associated Proteins; Plant Leaves; Recombinant Proteins; Signal Transduction; Sphingolipids	2020
Differential Effects of 2-Hydroxypropyl-Cyclodextrins on Lipid Accumulation in International journal of molecular sciences, 2020, Jan-30, Volume: 21, Issue:3 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; CHO Cells; Cholesterol; Cricetulus; Cyclodextrins; Lysosomes; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Sphingolipids	2020
Different Trafficking Phenotypes of Niemann-Pick C1 Gene Mutations Correlate with Various Alterations in Lipid Storage, Membrane Composition and Miglustat Amenability. International journal of molecular sciences, 2020, Mar-19, Volume: 21, Issue:6 Topics: 1-Deoxynojirimycin; Cells, Cultured; Cholesterol; Fibroblasts; Genotype; Humans; Membrane Microdomains; Membrane Proteins; Mutation; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Phenotype; Protein Transport; Sphingolipids	2020
Long-term survival outcomes of patients with Niemann-Pick disease type C receiving miglustat treatment: A large retrospective observational study. Journal of inherited metabolic disease, 2020, Volume: 43, Issue:5 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Aged; Aged, 80 and over; Child; Child, Preschool; Enzyme Inhibitors; Female; Humans; Infant; Infant, Newborn; Internationality; Male; Middle Aged; Niemann-Pick Disease, Type C; Registries; Retrospective Studies; Survival Analysis; Treatment Outcome; Young Adult	2020
Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry. Orphanet journal of rare diseases, 2020, 04-25, Volume: 15, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Enzyme Inhibitors; Female; Humans; Male; Niemann-Pick Disease, Type C; Prospective Studies; Registries; Treatment Outcome	2020
Long-term normalization of cognitive and psychopathological alterations in a juvenile Niemann-Pick type C case. Neurodegenerative disease management, 2020, Volume: 10, Issue:2 Topics: 1-Deoxynojirimycin; Adolescent; Child; Cognition; Disease Progression; Early Diagnosis; Female; Glycoside Hydrolase Inhibitors; Humans; Longitudinal Studies; Niemann-Pick Disease, Type C	2020
Novel compound heterozygous mutation in Journal of genetics, 2020, Volume: 99 Topics: 1-Deoxynojirimycin; Adult; Apathy; Brain; Cognitive Dysfunction; Corpus Callosum; Deglutition Disorders; Female; High-Throughput Nucleotide Sequencing; Humans; Intracellular Signaling Peptides and Proteins; Language Disorders; Magnetic Resonance Imaging; Mutation, Missense; Niemann-Pick C1 Protein; Niemann-Pick Disease, Type C; Vesicular Transport Proteins	2020
Characterization of Dysphagia and Longitudinal Changes in Swallowing Function in Adults with Niemann-Pick Disease Type C Treated with Miglustat. Dysphagia, 2021, Volume: 36, Issue:3 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Deglutition; Deglutition Disorders; Enzyme Inhibitors; Humans; Neurodegenerative Diseases; Niemann-Pick Disease, Type C; Retrospective Studies	2021
Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience. Molecular genetics & genomic medicine, 2020, Volume: 8, Issue:10 Topics: 1-Deoxynojirimycin; Adolescent; Central Nervous System; Child; Child, Preschool; Drug Tolerance; Female; Gangliosidosis, GM1; Glucosyltransferases; Glycoside Hydrolase Inhibitors; Humans; Infant; Male	2020
Association of Miglustat With Swallowing Outcomes in Niemann-Pick Disease, Type C1. JAMA neurology, 2020, 12-01, Volume: 77, Issue:12 Topics: 1-Deoxynojirimycin; Adolescent; Child; Child, Preschool; Cohort Studies; Deglutition; Deglutition Disorders; Enzyme Inhibitors; Female; Humans; Male; Niemann-Pick Disease, Type C; Pneumonia, Aspiration	2020
Early detection of Niemann-pick disease type C with cataplexy and orexin levels: continuous observation with and without Miglustat. Orphanet journal of rare diseases, 2020, 09-29, Volume: 15, Issue:1 Topics: 1-Deoxynojirimycin; Cataplexy; Female; Humans; Male; Niemann-Pick Disease, Type C; Orexins	2020
Gender-Specific Effects of Two Treatment Strategies in a Mouse Model of Niemann-Pick Disease Type C1. International journal of molecular sciences, 2021, Mar-03, Volume: 22, Issue:5 Topics: 1-Deoxynojirimycin; 2-Hydroxypropyl-beta-cyclodextrin; Animals; Cyclodextrins; Disease Models, Animal; Drug Therapy, Combination; Female; Male; Mice; Mice, Inbred BALB C; Niemann-Pick Disease, Type C; Pregnanolone	2021
Possible genotype-phenotype correlations in Niemann-Pick type C patients and miglustat treatment. Ideggyogyaszati szemle, 2021, Mar-30, Volume: 74, Issue:3-4 Topics: 1-Deoxynojirimycin; Genetic Association Studies; Humans; Mutation; Niemann-Pick Disease, Type C	2021
Inhibitors of Protein Glycosylation Are Active against the Coronavirus Severe Acute Respiratory Syndrome Coronavirus SARS-CoV-2. Viruses, 2021, 04-30, Volume: 13, Issue:5 Topics: 1-Deoxynojirimycin; A549 Cells; Animals; Antiviral Agents; Chlorocebus aethiops; COVID-19 Drug Treatment; Drug Repositioning; Glycoside Hydrolase Inhibitors; Glycosylation; HEK293 Cells; Humans; Indolizines; SARS-CoV-2; Spike Glycoprotein, Coronavirus; Vero Cells; Virus Release	2021
Cytokine profile and cholesterol levels in patients with Niemann-Pick type C disease presenting neurological symptoms: in vivo effect of miglustat and in vitro effect of N-acetylcysteine and coenzyme Q10. Experimental cell research, 2022, 07-15, Volume: 416, Issue:2 Topics: 1-Deoxynojirimycin; Acetylcysteine; Antioxidants; Cholesterol; Cytokines; Enzyme Inhibitors; Humans; Inflammation; Niemann-Pick Disease, Type C; Ubiquinone	2022
Cost-effectiveness of miglustat versus symptomatic therapy of Niemann-Pick disease type C. International journal of clinical pharmacy, 2022, Volume: 44, Issue:6 Topics: 1-Deoxynojirimycin; Adolescent; Cost-Benefit Analysis; Enzyme Inhibitors; Humans; Niemann-Pick Disease, Type C	2022
Could miglustat be a potential candidate in the treatment of action myoclonus renal failure syndrome? Acta neurologica Belgica, 2023, Volume: 123, Issue:6 Topics: 1-Deoxynojirimycin; Humans; Myoclonic Epilepsies, Progressive; Myoclonus	2023
Flow chemistry based catalytic hydrogenation for improving the synthesis of 1-deoxynojirimycin (DNJ) from an l-sorbose derived precursor. Carbohydrate research, 2023, Volume: 529 Topics: 1-Deoxynojirimycin; Hydrogenation; Sorbose	2023
Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study. Orphanet journal of rare diseases, 2023, 07-21, Volume: 18, Issue:1 Topics: 1-Deoxynojirimycin; Adolescent; Adult; Child; Child, Preschool; Female; Humans; Infant; Nervous System Diseases; Niemann-Pick Disease, Type C; Pregnancy; Retrospective Studies; Young Adult	2023