alpha-aminopyridine has been researched along with lumacaftor in 212 studies
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 1 (0.47) | 29.6817 |
| 2010's | 144 (67.92) | 24.3611 |
| 2020's | 67 (31.60) | 2.80 |
| Authors | Studies |
|---|---|
| Retsch-Bogart, GZ | 1 |
| Accurso, FJ; Aitken, ML; Amin, RS; Ashlock, MA; Ballmann, M; Botfield, M; Boyle, MP; Bronsveld, I; Campbell, PW; Clancy, JP; De Boeck, K; Donaldson, SH; Dorkin, HL; Dunitz, JM; Durie, PR; Jain, M; Konstan, MW; Leonard, A; McCoy, KS; Moss, RB; Ordoñez, CL; Pilewski, JM; Rosenbluth, DB; Rowe, SM; Rubenstein, RC; Schechter, MS; Spencer-Green, GT; Vernillet, L; Wisseh, S; Yen, K | 1 |
| Ashlock, M; Burton, B; Decker, CJ; Frizzell, RA; Grootenhuis, PD; Hadida, S; McCartney, J; Miller, M; Negulescu, PA; Olson, ER; Stack, JH; Straley, KS; Van Goor, F; Wine, JJ | 1 |
| Vij, N | 1 |
| Aleksandrov, AA; Cui, L; Dokholyan, NV; He, L; Jensen, T; Kota, P; Riordan, JR | 1 |
| Bertoncini, E; Colomb-Lippa, D | 1 |
| Alexander, RT; Berrini, M; Chu, CY; Cordat, E; King, JC | 1 |
| Fox, JL | 1 |
| Bagdany, M; Beekman, JM; Dekkers, JF; Hegedus, T; Kurth, M; Lukacs, GL; Okiyoneda, T; Roldan, A; Simon, A; Soya, N; Veit, G; Verkman, AS; Xu, H | 1 |
| Galietta, LJ | 1 |
| Rowe, SM; Verkman, AS | 1 |
| Bartlett, MC; Clarke, DM; Loo, TW | 1 |
| Amaral, MD; Correia, AR; Da Paula, AC; Farinha, CM; Gomes, CM; Henriques, BJ; Hirst, S; King-Underwood, J; Roxo-Rosa, M; Sousa, M; Williams, J | 1 |
| Cyr, DM; De La Rosa, O; Grove, DE; Hoffman, BJ; Houck, SA; Ren, HY; Sopha, P; Van Goor, F | 1 |
| Durieu, I; Durupt, S; Nove Josserand, R | 1 |
| Clarke, DM; Loo, TW | 2 |
| Doe, SJ; Lane, MA | 1 |
| Baskin, B; Bear, CE; Gonska, T; Huan, LJ; Janahi, IA; Molinski, SV; Ray, PN | 1 |
| Ahmadi, S; Bear, CE; Beekman, J; Chung, TE; Dekkers, JF; Du, K; Eckford, PD; Gonska, T; Ip, W; Li, C; Molinski, S; Pasyk, S; Ramjeesingh, M; Yeger, H | 1 |
| Finkbeiner, WE; Lukacs, GL; Phuan, PW; Roldan, A; Tan, J; Veit, G; Verkman, AS | 1 |
| Baroni, D; Moran, O; Svensson, A; Zegarra-Moran, O | 1 |
| Bompadre, SG; Kopeikin, Z; Yang, HY; Yuksek, Z | 1 |
| Arora, K; Moon, C; Naren, AP; Penmatsa, H; Ren, A; Sinha, C; Yarlagadda, S; Zhang, W | 1 |
| Becq, F; Boinot, C; Ferru-Clément, R; Jollivet Souchet, M | 1 |
| Tümmler, B | 2 |
| Bell, SC; Boyle, MP; Huang, X; Konstan, MW; McColley, SA; Patel, NR; Rietschel, E; Rodman, D; Rowe, SM; Waltz, D | 1 |
| Chiba, P; Freissmuth, M; Rudashevskaya, EL; Stockner, T; Trauner, M | 1 |
| Becq, F; Callebaut, I; Jollivet Souchet, M; Prasad, R; Sharma, H | 1 |
| Chen, JH | 1 |
| Baroni, D; Moran, O; Zegarra-Moran, O | 1 |
| Kumar, S; Shankar, A; Tana, A | 1 |
| Eldredge, LC; Ramsey, BW | 1 |
| Antigny, F; Becq, F; Buscaglia, P; Frieden, M; Mignen, O; Norez, C; Philippe, R | 1 |
| Ma, T; Yang, H | 1 |
| Boyle, MP; Cipolli, M; Colombo, C; Davies, JC; De Boeck, K; Elborn, JS; Flume, PA; Huang, X; Konstan, MW; Marigowda, G; McColley, SA; McCoy, K; McKone, EF; Munck, A; Ramsey, BW; Ratjen, F; Rowe, SM; Wainwright, CE; Waltz, D | 1 |
| Mayor, S | 1 |
| Amaral, MD; Barros, P; Dias-Alves, Â; Loureiro, CA; Matos, AM; Matos, P; Pereira, JF; Uliyakina, I | 1 |
| Barnaby, R; Coutermarsh, B; Hogan, D; Stanton, BA | 1 |
| Akkermans, R | 1 |
| Barry, PJ; Jones, AM | 1 |
| Cebotaru, L; Grover, R; Guggino, WB; Handa, JT; Lopes Pacheco, M; Rapino, D; Sabirzhanova, I | 1 |
| Marshall, SG; Ramsey, BW; Rowe, SM; Solomon, GM | 1 |
| Amaral, MD; Awatade, NT; Clarke, LA; Farinha, CM; Mendes, K; Pastor, J; Ramos, MM; Solé, A; Uliyakina, I | 1 |
| Nau, JY | 1 |
| Actis, M; Arora, K; Clancy, JP; Frizzell, R; Fujii, N; Lin, S; Moon, CS; Naren, AP; Sinha, C; Woodroofe, K; Yarlagadda, S; Zhang, W; Ziady, AG | 1 |
| Finkbeiner, WE; Lukacs, GL; Phuan, PW; Tan, JA; Veit, G; Verkman, AS | 1 |
| Carlier, J; Kupchick, T; Larder, A; McCarty, J; Powers, M; Thomas, J; Virant-Young, D; Woiderski, S | 1 |
| Bali, V; Bebok, Z; Fu, L; Guroji, P; Lazrak, A; Matalon, S | 1 |
| Frizzell, RA; Hu, J; Larsen, MB; Watkins, SC | 1 |
| Ong, T; Ramsey, BW | 2 |
| Kuk, K; Taylor-Cousar, JL | 1 |
| Billet, A; Carlile, GW; Dejgaard, K; Goepp, J; Hanrahan, JW; Luo, Y; Matthes, E; Robert, R; Thomas, DY | 1 |
| Chan, HC; Chen, J; Chen, Y; Dong, ZW; Peng, YZ; Ruan, YC; Tsang, LL; Zhou, T | 1 |
| Azvolinsky, A | 1 |
| Brewington, JJ; Clancy, JP; McPhail, GL | 1 |
| Capurro, V; Galietta, LJ; Gianotti, A; Moran, O; Scudieri, P; Zegarra-Moran, O | 1 |
| Anzaldi, M; Galietta, LJ; Mazzei, M; Mazzei, MT; Nieddu, E; Pedemonte, N; Pollarolo, B; Schenone, S | 1 |
| Jeon, DK; Jo, S; Khloya, P; Kumar, S; Lee, HK; Namkung, W; Park, J; Seo, Y; Sharma, PK | 1 |
| Desch, M | 1 |
| Quon, BS; Rowe, SM | 1 |
| Beekman, JM; de Winter-de Groot, KM; Dekkers, JF; Gogorza Gondra, RA; Janssens, HM; Kruisselbrink, E; van der Ent, CK; Vonk, AM | 1 |
| Atlante, A; Bobba, A; Casavola, V; Favia, M; Guerra, L; Reshkin, SJ | 1 |
| Apaja, PM; Bidaud-Meynard, A; Guo, J; Hartman, JL; Icyuz, M; Lin, ST; Lukacs, GL; Oliver, K; Perdomo, D; Sorscher, EJ; Veit, G | 1 |
| Bali, V; Bebok, Z; Guroji, P; Lazrak, A; Matalon, S | 1 |
| Naren, AP; Strokes, DC; Zhang, W; Zhang, X; Zhang, YH | 1 |
| Boyle, MP; Elborn, JS; Huang, X; Konstan, MW; Marigowda, G; Ramsey, BW; Wainwright, CE; Waltz, D | 1 |
| Gulland, A | 1 |
| Beekman, JM; Berkers, G; Bronsveld, I; Clevers, H; de Jonge, HR; de Rijke, YB; de Winter-de Groot, KM; Dekkers, JF; Escher, JC; Heijerman, HG; Houwen, RH; Janssens, HM; Kruisselbrink, E; Majoor, CJ; Nieuwenhuis, EE; van de Graaf, EA; van der Ent, CK; Vleggaar, FP; Vonk, A | 1 |
| Deeks, ED | 1 |
| Chaudhry, IG; Cholon, DM; Cyr, DM; Das, J; Dokholyan, NV; Gentzsch, M; Houck, SA; Quinney, NL; Randell, SH; Ren, HY; Sopha, P | 1 |
| Hussar, DA | 1 |
| Bear, CE; Casavola, V; Laselva, O; Molinski, S | 2 |
| Azad, MA; Baker, MA; Bergen, PJ; Cooper, MA; Doi, Y; Han, ML; Huang, JX; Li, J; Muller, MT; Schneider, EK; Tony Zhou, Q; Velkov, T; Wang, J | 1 |
| Keating, D; Kotsimbos, T; Li, J; Reyes-Ortega, F; Schneider, EK; Velkov, T; Wilson, JW | 1 |
| Baksh, MM; Finn, MG; Heidary, DK; Richards, CI; Zhang, Z | 1 |
| Cai, Z; Ford, RC; Li, H; Meng, X; Rimington, TL; Sheppard, DN; Wang, X; Wang, Y; Wrennall, JA | 1 |
| Aitken, ML; Godfrey, EM; Heltshe, SL; Josephy, T; Taylor-Cousar, JL | 1 |
| Egan, ME | 1 |
| Barry, P; Horsley, A | 1 |
| Brouillette, CG; Chong, PA; Dawson, JE; Forman-Kay, JD; Hudson, RP; Millen, L; Thomas, PJ; Yang, Z | 1 |
| Apergis-Schoute, J; Cotel, F; Fletcher, LN; Kalita-de Croft, S; Williams, SR | 1 |
| Casciaro, R; Costa, S; Dang, P; Majo, F; Ros, M | 1 |
| Amenduni, M; Fabris, L; Fiorotto, R; Mariotti, V; Spirli, C; Strazzabosco, M | 1 |
| AbuArish, A; Cantin, AM; Cloutier, A; Cosa, G; Greene, LE; Hanrahan, JW; Matthes, E; Robert, R; Thomas, DY; Turner, MJ; Wong, FH | 1 |
| Chitre, A; Crotti, L; Gnecchi, M; Lua, CH; Mehta, A; Mura, M; Ramachandra, CJA; Schwartz, PJ; Shim, W; Singh, P; Wong, P | 1 |
| Delisle, BP; January, CT | 1 |
| Ashare, A; Barnaby, R; Berwin, B; Hampton, TH; Koeppen, K; Nymon, A; Stanton, BA | 1 |
| Li, J; Reyes-Ortega, F; Schneider, EK; Velkov, T | 1 |
| Adam, D; Brochiero, E; Lafayette, SL; Maillé, É; McKay, G; Messaoud, H; Nguyen, D; Ruffin, M | 1 |
| Aban, I; Boyles, SE; Chaudhry, IG; Chua, M; Gentzsch, M; Guimbellot, JS; Jaspers, I; Leach, JM; Quinney, NL | 1 |
| Adolphe, C; El-Seedy, A; Farhat, R; Kitzis, A; Ladevèze, V; Norez, C; Pasquet, MC; Talbot, H | 1 |
| DiMango, E; Hopkins, BD; Kitur, K; Parsons, R; Prince, A; Riquelme, SA; Wolfe, AL | 1 |
| Arkel, M; Cichero, E; Damonte, G; Fossa, P; Galietta, LJV; Liessi, N; Millo, E; Paccagnella, M; Pedemonte, N; Pesce, E; Salis, A; Tasso, B; Tomati, V | 1 |
| Brochiero, E; Maillé, É; Rousseau, S; Roussel, L; Ruffin, M | 1 |
| Bruchez, MP; Fisher, GW; Mun, F; Naganbabu, M; Perkins, LA; Schmidt, BF | 1 |
| Bell, SC; Wood, ME | 1 |
| Liu, H; Luo, D; Luo, K | 1 |
| Backstrom, J; Brewington, JJ; Clancy, JP; Feldman, A; Kramer, EL; Lu, LJ; Moncivaiz, JD; Ostmann, AJ; Zhu, X | 1 |
| Burgener, EB; Moss, RB | 1 |
| Brewington, JJ; Clancy, JP; Harkness, B; McCarthy, C; Trapnell, BC | 1 |
| Bear, CE; Laforet, M; Laselva, O; MacKinnon, SS; Molinski, SV; Morayniss, LD; Shahani, VM; Subramanian, AS; Windemuth, A; Woollard, G | 1 |
| Gentzsch, M; Mall, MA | 1 |
| Cebotaru, L; Liu, Q; Yanda, MK | 1 |
| Ahmadi, S; Bear, CE; Chin, S; Elmallah, S; Hamilton, CM; Hung, M; Toutah, K; Viirre, RD; Won, A; Wu, YS; Yang, D; Yip, CM; Young, RN | 1 |
| Bergbower, EAS; Boinot, C; Cebotaru, L; Guggino, WB; Liu, Q; Sabirzhanova, I; Yanda, MK | 1 |
| Cantin, AM; Hanrahan, JW; Matthes, E | 1 |
| Cai, Z; Cutting, GR; Davis, EF; Evans, TA; Han, ST; Hong, JS; Joynt, AT; Lu, Z; McCague, AF; Pellicore, MJ; Rab, A; Raraigh, KS; Sheppard, DN; Sorscher, EJ | 1 |
| Carlile, GW; Hanrahan, JW; Liao, J; Matthes, E; Miyamoto, C; Radinovic, S; Robert, R; Thomas, DY; Yang, Q | 1 |
| Nevitt, SJ; Patel, S; Sinha, IP; Southern, KW | 2 |
| Barry, PJ; Jones, AM; Mitchell, RM | 1 |
| Amaral, MD; Barros, P; Faria, M; Gomes-Duarte, A; Jordan, P; Matos, AM; Matos, P | 1 |
| Caskey, RN; Dowell, ML; Hung, YT; Sharma, D; Touchette, DR; Xing, S | 1 |
| Boyles, SE; Cholon, DM; Gentzsch, M; Martino, MEB; Quinney, NL; Ribeiro, CMP | 1 |
| Bonk, MP; Hadjiliadis, D; Rey, MM | 1 |
| Bailly, C; Chedevergne, F; Edelman, A; Flament, T; Hatton, A; Hinzpeter, A; Le Bourgeois, M; Masson, A; Pranke, I; Sermet-Gaudelus, I; Urbach, V | 1 |
| Adewale, AT; Ashtekar, AR; Brand, JD; Harrod, KS; Lazrak, A; Matalon, S; Rowe, SM; Shei, RJ; Tipper, JL; Trombley, JE; Yu, Z | 1 |
| Hwang, TC; Yeh, JT; Yu, YC | 1 |
| Barrio, M; Blanco-Aparicio, M; Cols-I-Roig, M; Delgado-Pecellín, I; Diab-Cáceres, L; Fernández, O; García-Clemente, MM; Girón-Moreno, RM; Gómez-de-Terreros-Caro, FJ; González, M; López-Neyra, A; Luna-Paredes, C; Maiz, L; Mondéjar-López, P; Palou-Rotger, A; Pastor-Sanz, MT; Quintana-Gallego, E; Ruiz-de-Valbuena, M | 1 |
| Beekman, JM; Braakman, I; de Jonge, HR; Egmond, MR; Kleizen, B; Kruisselbrink, E; van der Ent, CK; van der Sluijs, P; van Willigen, M; Vonk, AM; Yeoh, HY | 1 |
| Garbuzenko, OB; Kbah, N; Kuzmov, A; Minko, T; Pogrebnyak, N; Pozharov, V | 1 |
| Flume, PA | 1 |
| Li, C; Liu, F; Marigowda, G; McColley, SA; McNamara, JJ; Owen, CA; Sawicki, GS; Stiles, D; Tian, S; Waltz, D; Wang, LT | 1 |
| Amaral, MD; Bertuzzo, CS; Kmit, A; Leite, GS; Marson, FAL; Pereira, SV; Ribeiro, AF; Ribeiro, JD; Servidoni, MF; Vinagre, AM | 1 |
| Baroni, D; Ferrera, L; Moran, O | 1 |
| Anglès, F; Balch, WE; Hutt, DM | 1 |
| Bijvelds, MJC; Bose, SJ; Bot, AGM; Cai, Z; de Jonge, HR; Liu, J; Sheppard, DN; Wang, Y | 1 |
| Bratcher, PE; Chioccioli, M; Cicuta, P; Feriani, L; Kotar, J | 1 |
| Baldelli, S; Cattaneo, D; Clementi, E; Colombo, C; Cozzi, V; Faelli, NML; Fusi, M; Russo, M | 1 |
| Beekman, JM; Chen, KG; Zheng, W; Zhong, P | 1 |
| Burki, TK | 1 |
| Bergbower, EAS; Cebotaru, L; Guggino, WG; Liu, Q; Sabirzhanova, I; Yanda, M | 1 |
| Beiersdorf, N; Groten, T; Jaudszus, A; Lorenz, M; Mainz, JG; Michl, RK; Schneider, U | 1 |
| Alper, CJ; Bacon, R; Greenwood, BC; Jeffrey, PL; Lenz, K; Stevens, K; Tesell, MA | 1 |
| Akshintala, VS; Cebotaru, L; Cutting, GR; Dezube, R; Faghih, M; Jennings, MT; Kamal, A; Lechtzin, N; Merlo, CA; Singh, VK; West, NE; Whitcomb, DC | 1 |
| Jain, M; Sala, MA | 1 |
| Braakman, I; Hartman, JL; Hong, JS; Ignatova, Z; Kesterson, RA; Kirk, KL; Maya, J; Mijnders, M; Oliver, KE; Rab, A; Rauscher, R; Sabusap, CM; Sorscher, EJ; Wang, W; Wolpert, MJ | 1 |
| Cuyx, S; De Boeck, K | 1 |
| Amico, G; Baroni, D; Brandas, C; Moran, O | 1 |
| Ehrhardt, A; Hong, JS; Joshi, D; Sorscher, EJ | 1 |
| Mall, MA; Mayer-Hamblett, N; Rowe, SM | 1 |
| Deber, CM; Hartmann, A; Krainer, G; Ravamehr-Lake, D; Schenkel, M; Schlierf, M | 1 |
| Clifton, I; Etherington, C; Holbrook, J; Jarosz-Griffiths, HH; Lara-Reyna, S; Martinon, F; McDermott, MF; Mehta, A; Peckham, D; Savic, S; Scambler, T; Spoletini, G; Whitaker, P; Wong, CH | 1 |
| Bensalah, M; Carotti, M; Fancello, I; Richard, I; Risato, G; Sandonà, D; Scano, M; Soardi, M | 1 |
| Hanrahan, JW; Luo, Y; Thomas, DY; Turner, MJ | 1 |
| Daniels, MLA; Ebert, CS; Farzal, Z; Kimple, AJ; Lee, SE; Senior, BA; Thorp, BD; Zanation, AM | 1 |
| Cottrill, KA; Farinha, CM; McCarty, NA | 1 |
| Aller, SG; Bacsa, J; Breton, GW; Doiron, JE; Le, CA; Martin, KL; Turlington, M | 1 |
| Kessler, L | 1 |
| Fiore, M; Moran, O; Picco, C | 1 |
| Boyle, KG; Dhillon, SS; Guenette, JA; Mitchell, RA; Quon, BS; Ramsook, AH; Wilcox, PG | 1 |
| Ballmann, M; Glass, A; Holl, R; Prinz, N | 1 |
| Keeling, KM; Rowe, SM; Sharma, J | 1 |
| Clancy, JP; Donaldson, SH; Khan, U; Ratjen, F; Rowe, SM; Sagel, SD; Shaw, M | 1 |
| Mall, MA; Naisbitt, DJ; Ogese, MO; Roehmel, JF; Rohrbach, A | 1 |
| Aureli, M; Bassi, R; Cabrini, G; Chiricozzi, E; Dechecchi, MC; Lippi, G; Loberto, N; Mancini, G; Mauri, L; Olioso, D; Pedemonte, N; Pesce, E; Schiumarini, D; Sonnino, S; Tamanini, A | 1 |
| Amaral, MD; Carlile, GW; Hanrahan, JW; Lopes-Pacheco, M; Pedemonte, N; Silva, IAL; Sondo, E; Thomas, DY; Turner, MJ | 1 |
| Afonso, S; Amaral, MD; Botelho, HM; da Paula, AC; Farinha, CM; Felício, V; Lobo, MJ; Uliyakina, I | 1 |
| Aissat, A; Bizard, L; Decrouy, X; Degrugillier, F; Fanen, P; Jiang, C; Prulière-Escabasse, V; Rotin, D; Simon, S; Simonneau, B | 1 |
| Finkbeiner, WE; Haggie, PM; Janahi, IA; Nielson, DW; Phuan, PW; Rivera, AA; Tan, JA; Thomas, MM; Verkman, AS | 1 |
| Amaral, MD; Botelho, HM; Centeio, R; Clarke, LA; Doušová, T; Farinha, CM; Holubová, A; Hwang, TC; Kunzelmann, K; Railean, V; Ramalho, S; Silva, IAL; Valášková, I; Yeh, JT | 1 |
| Beaufils, F; Bui, S; Burgel, PR; Delhaes, L; Enaud, R; Fayon, M; Lamireau, T; Mas, E; Maumus, P; Mittaine, M; Tétard, C | 1 |
| Iovinelli, D; Prischi, F; Santucci, A; Spiga, O; Trezza, A | 1 |
| Ackerman, MJ; Hamrick, SK; John Kim, CS; O'Hare, BJ; Tester, DJ; Ye, D | 1 |
| Casement, J; Delpiano, L; Gray, MA; Lin, J; Onuora, JC; Saint-Criq, V | 1 |
| Cichero, E; Damonte, G; Millo, E; Parodi, A; Pedemonte, N; Pesce, E; Righetti, G; Rusnati, M; Salis, A; Tasso, B; Tomati, V; Urbinati, C | 1 |
| Casale, M; Cichero, E; Fossa, P; Liessi, N; Millo, E; Pedemonte, N; Righetti, G; Salis, A; Tasso, B; Tonelli, M | 1 |
| Bos, LDJ; Kemper, EM; Maitland-van der Zee, AH; Majoor, CJ; Mathôt, RAA; Neerincx, AH; van der Meer-Vos, M; Vonk, SEM | 1 |
| Duncan, M; Ellis, M; Harris, RA; Jha, L; Keating, D; Kotsimbos, T; Tian, S; Urquhart, DS; Wilson, J; You, X | 1 |
| Lechtzin, N; Montemayor, K | 1 |
| Dave, K; Davies, JC; Dobra, R; Matthews, J; Saunders, C; Scott, S; Simmonds, NJ | 1 |
| Bardin, E; Berhal, F; Chevalier, B; Golec, A; Gravier-Pelletier, C; Hayes, K; Hinzpeter, A; Pastor, A; Pranke, I; Prestat, G; Semeraro, M; Sermet-Gaudelus, I | 1 |
| Battezzati, A; Bisogno, A; Colombo, C; Daccò, V; Foppiani, A; Gambazza, S; Giana, A; Leone, A; Mari, A; Nazzari, E | 1 |
| Chen, J; Conrath, K; Falk Libby, E; Li, Y; Mutyam, V; Peng, N; Rowe, SM; Sharma, J; Singh, AK; Tang, LP | 1 |
| Amato, F; Castaldo, G; Castellani, C; Ferrari, B; Galietta, LJV; Taccetti, G; Terlizzi, V | 1 |
| Braakman, I; Frizzell, R; Grudniewska, M; Hillenaar, T; Kleizen, B; Kooijman, L; Mijnders, M; Peters, F; Peters, KW; Thomas, A; van der Sluijs, P; van Willigen, M | 1 |
| Course, CW; Doull, I; Edmondson, C | 1 |
| Bailly, B; Day, CJ; Dirr, L; Guillon, P; Haselhorst, T; Jen, FE; Jennings, MP; Mak, J; Spillings, BL; von Itzstein, M | 1 |
| Franceschelli, S; Pascale, M; Pecoraro, M | 1 |
| Chandra, S; Joshi, T; Pundir, H | 1 |
| Chilvers, M; Cornell, AG; Hoppe, JE; McColley, SA; McNamara, JJ; Owen, CA; Ratjen, F; Tian, S; Zahigian, R | 1 |
| Harwood, KH; Jarnicki, A; McQuade, RM; Schneider-Futschik, EK | 1 |
| Bailly-Botuha, C; Berteloot, L; Chedevergne, F; Cornet, M; Dana, J; Debray, D; Drummond, D; Girard, M; Le Bourgeois, M; Nguyen-Khoa, T; Schneider-Futschik, EK; Sermet-Gaudelus, I | 1 |
| Chróinín, MN; Croinin, K; Crowley, J; Mullane, D | 1 |
| Cebotaru, L; Yanda, MK | 1 |
| Bruegel, M; Habler, K; Kalla, AS; Nährig, S; Paal, M; Rychlik, M; Teupser, D; Vogeser, M | 1 |
| Baroni, D; Brandas, C; Cichero, E; Ludovico, A; Millo, E; Moran, O; Parodi, A | 1 |
| Baatallah, N; Callebaut, I; Chevalier, B; Décout, JL; Edelman, A; Elbahnsi, A; Hinzpeter, A; Mornon, JP; Pranke, I; Sermet-Gaudelus, I; Servel, N; Zelli, R | 1 |
| Borrelli, A; Capurro, V; Galietta, LJV; Musante, I; Pedemonte, N; Renda, M; Scudieri, P; Venturini, A | 1 |
| Amaral, MD; Pinto, MC; Quaresma, MC; Railean, V; Ramalho, SS; Silva, IAL | 1 |
| Kondapalli, VGCS; Mullangi, S; Panchakarla, RK; Ravi, PR | 1 |
| Chen, J; Fiedorczuk, K | 1 |
| Zhang, B; Zhang, S | 1 |
| Burgel, PR; Campredon, A; Chassagnon, G; Vakalopoulou, M | 1 |
| Borowitz, D; Clancy, JP; Gabel, ME; Gelfond, D; Roach, C; Rowe, SM; Sagel, SD; Wang, H | 1 |
| Jensen, JH; Leo-Hansen, C; Olesen, HV; Pedersen, CL; Philipsen, LKD; Rysgaard, UK; Sørensen, L | 1 |
| Armirotti, A; Baldassari, S; Bandiera, T; Braccia, C; Breckels, LM; Capurro, V; Christopher, JA; Crook, OM; Liessi, N; Lilley, KS; Pedemonte, N; Queiroz, RML; Tomati, V | 1 |
| Edwards, C; Lee, T; Naisbitt, DJ; Nissenbaum, C; Ogese, MO; Pirmohamed, M; Semic-Jusufagic, A; Wilkinson, M | 1 |
| Neeland, MR; Ranganathan, S; Shanthikumar, S | 1 |
| Bihouee, T; Bonnel, AS; Chedevergne, F; Ciciriello, F; Cornet, M; Kelly, M; Lebourgeois, M; Lucidi, V; Marguet, C; Reix, P; Robin, G; Roy, V; Sermet-Gaudelus, I; Stoven, V | 1 |
| Barth, S; Naehrlich, L; Westhoff, J | 1 |
| Amaral, MD; Balogh, I; Bene, Z; Fejes, Z; Macek, M; Nagy, A; Nagy, B; Pócsi, M | 1 |
| Bandiera, T; Barraja, P; Barreca, M; Bivacqua, R; Borrelli, A; Buccirossi, M; Galietta, LJV; Genovese, M; Guidone, D; Montalbano, A; Musante, I; Raimondi, MV; Renda, M; Scudieri, P; Spanò, V; Venturini, A | 1 |
| Beringer, P; Hong, E; Shi, A | 1 |
| Dixon, B; Evans, CN; Girard, C; Henager, ME; Herring, C; Johnson, QR; Lester, A; Mitchell, A; Poulos, J; Ramsdell, H; Reber, C; Sandman, M; Spinney, A; Turlington, M | 1 |
| Bournissen, FG; Lubovich, S; Rodríguez, E; Rodríguez, V; Teper, A; Zaragoza, S | 1 |
34 review(s) available for alpha-aminopyridine and lumacaftor
| Article | Year |
|---|---|
Update on new pulmonary therapies.
Topics: Aminophenols; Aminopyridines; Anti-Infective Agents; Anti-Inflammatory Agents; Benzodioxoles; Biomarkers; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genetic Markers; Genetic Therapy; Humans; Mutation; Oxadiazoles; Quinolones; Respiratory System Agents; Saline Solution, Hypertonic | 2009 |
Managing the underlying cause of cystic fibrosis: a future role for potentiators and correctors.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Clinical Trials as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Humans; Molecular Targeted Therapy; Mutation; Quinolones | 2013 |
Cystic fibrosis transmembrane regulator correctors and potentiators.
Topics: Aminophenols; Aminopyridines; Bayes Theorem; Benzodioxoles; Chloride Channels; Clinical Trials, Phase II as Topic; Clinical Trials, Phase III as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Discovery; Humans; Mutation; Quinolones; Randomized Controlled Trials as Topic; Technology, Pharmaceutical | 2013 |
[Therapeutic update in cystic fibrosis].
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation; Oxadiazoles; Quinolones | 2014 |
Pharmacological correction of misfolding of ABC proteins.
Topics: Aminopyridines; Animals; ATP-Binding Cassette Transporters; Benzodioxoles; Cholestasis, Intrahepatic; Clinical Trials as Topic; Cystic Fibrosis; Drug Discovery; Humans; Protein Binding; Protein Folding; Protein Transport; Proteostasis Deficiencies; Small Molecule Libraries | 2014 |
Cystic fibrosis--what are the prospects for a cure?
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genetic Therapy; Humans; Quinolones | 2014 |
F508del-cystic fibrosis transmembrane regulator correctors for treatment of cystic fibrosis: a patent review.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Design; Humans; Mutation; Patents as Topic; Quinolones | 2015 |
Breakthrough therapies: Cystic fibrosis (CF) potentiators and correctors.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Clinical Trials as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Design; Homozygote; Humans; Mice; Molecular Targeted Therapy; Mutation; Protein Folding; Quinolones | 2015 |
Cystic Fibrosis: A Novel Pharmacologic Approach to Cystic Fibrosis Transmembrane Regulator Modulation Therapy.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; DNA; DNA Mutational Analysis; Humans; Mutation; Oxadiazoles; Quinolones | 2015 |
Update in Cystic Fibrosis 2014.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genetic Markers; Humans; Mucociliary Clearance; Quinolones; Treatment Outcome | 2015 |
Lumacaftor and ivacaftor in the management of patients with cystic fibrosis: current evidence and future prospects.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; DNA Mutational Analysis; Drug Combinations; Genetic Predisposition to Disease; Humans; Lung; Mutation; Patient Selection; Phenotype; Precision Medicine; Quinolones; Respiratory System Agents; Treatment Outcome | 2015 |
[Treatment of Cystic Fibrosis with CFTR Modulators].
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers, Tumor; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Dose-Response Relationship, Drug; Drug Combinations; Evidence-Based Medicine; Genetic Markers; Genetic Predisposition to Disease; Humans; Precision Medicine; Quinolones; Treatment Outcome | 2016 |
New and emerging targeted therapies for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Clinical Trials as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Discovery; Drug Therapy, Combination; Forced Expiratory Volume; Forecasting; Genetic Therapy; Homozygote; Humans; Molecular Targeted Therapy; Mutation; Oxadiazoles; Phosphodiesterase 5 Inhibitors; Practice Guidelines as Topic; Precision Medicine; Quinolones | 2016 |
Lumacaftor/ivacaftor combination for cystic fibrosis patients homozygous for Phe508del-CFTR.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Clinical Trials as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Humans; Mutation; Precision Medicine; Quinolones | 2016 |
Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Clinical Trials, Phase III as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Double-Blind Method; Drug Therapy, Combination; Female; Forced Expiratory Volume; Homozygote; Humans; Lung; Male; Middle Aged; Quinolones; Randomized Controlled Trials as Topic; Respiratory Function Tests; Treatment Outcome; Young Adult | 2016 |
Lumacaftor/Ivacaftor: A Review in Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Lung; Mutation; Quinolones | 2016 |
New Therapeutic Approaches to Modulate and Correct Cystic Fibrosis Transmembrane Conductance Regulator.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Molecular Targeted Therapy; Mutation; Quinolones | 2016 |
Cystic fibrosis transmembrane conductance regulator modulators: precision medicine in cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Clinical Trials, Phase II as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Humans; Indoles; Precision Medicine; Quinolones; Respiratory System Agents | 2018 |
Ion Channel Modulators in Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genotype; Humans; Indoles; Ion Channels; Precision Medicine; Quinolones | 2018 |
Correctors (specific therapies for class II CFTR mutations) for cystic fibrosis.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genetic Therapy; Humans; Indoles; Mutation; Phenylbutyrates; Quinolones; Randomized Controlled Trials as Topic | 2018 |
CFTR modulator therapy in patients with cystic fibrosis and an organ transplant.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Male; Molecular Targeted Therapy; Organ Transplantation; Quinolones; Young Adult | 2018 |
A systematic Cochrane Review of correctors (specific therapies for class II CFTR mutations) for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Humans; Indoles; Quinolones; Treatment Outcome | 2019 |
Pharmacological analysis of CFTR variants of cystic fibrosis using stem cell-derived organoids.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biological Assay; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genotype; Humans; Molecular Targeted Therapy; Mutation; Organoids; Pharmacogenomic Variants; Quinolones; Stem Cells | 2019 |
Treating the Underlying Cystic Fibrosis Transmembrane Conductance Regulator Defect in Patients with Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Clinical Trials, Phase II as Topic; Clinical Trials, Phase III as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genetic Therapy; Humans; Indoles; Mutation; Quinolones; Randomized Controlled Trials as Topic | 2019 |
Cystic fibrosis precision therapeutics: Emerging considerations.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Indoles; Precision Medicine; Quinolones | 2019 |
Cystic Fibrosis: Emergence of Highly Effective Targeted Therapeutics and Potential Clinical Implications.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Sodium Channel Blockers; Humans; Indoles; Molecular Targeted Therapy; Mucociliary Clearance; Mutation; Precision Medicine; Pyrazoles; Pyridines; Pyrrolidines; Quinolones | 2020 |
Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy: A Review for the Otolaryngologist.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chronic Disease; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Approval; Humans; Indoles; Mutation; Nasal Mucosa; Otolaryngologists; Pyrazoles; Pyridines; Pyrrolidines; Quinolones | 2020 |
The bidirectional relationship between CFTR and lipids.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Epithelial Cells; Humans; Lung; Membrane Lipids; Membrane Microdomains; Mutation; Protein Conformation; Protein Stability; Protein Transport; Quinolones; Structure-Activity Relationship | 2020 |
Pharmacological approaches for targeting cystic fibrosis nonsense mutations.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Codon, Nonsense; Cystic Fibrosis; Dose-Response Relationship, Drug; Humans; Indoles; Molecular Structure; Mutation; Pyrazoles; Pyridines; Pyrrolidines; Quinolones; Structure-Activity Relationship | 2020 |
Entering the era of highly effective modulator therapies.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Clinical Trials as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Humans; Indoles; Mutation; Pyrazoles; Pyridines; Pyrrolidines; Quinolones | 2021 |
Modulators of CFTR. Updates on clinical development and future directions.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Clinical Trials as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Development; Humans; Indoles; Quinolones | 2021 |
Cystic fibrosis transmembrane conductance regulator modulators for cystic fibrosis: a new dawn?
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genetic Variation; Humans; Indoles; Outcome Assessment, Health Care; Pyrazoles; Pyridines; Pyrrolidines; Quinolones | 2021 |
Anti-Inflammatory Influences of Cystic Fibrosis Transmembrane Conductance Regulator Drugs on Lung Inflammation in Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Anti-Inflammatory Agents; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Indoles; Inflammation; Ion Transport; Lung; Macrophages; Pneumonia; Quinolones; Signal Transduction | 2021 |
Drug-drug interactions involving CFTR modulators: a review of the evidence and clinical implications.
Topics: Aminopyridines; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Interactions; Humans; Mutation | 2023 |
6 trial(s) available for alpha-aminopyridine and lumacaftor
| Article | Year |
|---|---|
Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation.
Topics: Adolescent; Adult; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; DNA; DNA Mutational Analysis; Dose-Response Relationship, Drug; Double-Blind Method; Female; Follow-Up Studies; Homozygote; Humans; Male; Middle Aged; Mutation; Prospective Studies; Sweat Glands; Treatment Outcome; Young Adult | 2012 |
A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Base Sequence; Benzodioxoles; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Double-Blind Method; Drug Therapy, Combination; Female; Forced Expiratory Volume; Heterozygote; Homozygote; Humans; Male; Quinolones; Sequence Deletion; Sweat; Young Adult | 2014 |
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Double-Blind Method; Drug Therapy, Combination; Female; Forced Expiratory Volume; Homozygote; Hospitalization; Humans; Male; Middle Aged; Mutation; Quinolones; Young Adult | 2015 |
Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study.
Topics: Age Factors; Aminophenols; Aminopyridines; Benzodioxoles; Child, Preschool; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Female; Homozygote; Humans; Male; Quinolones | 2019 |
Short-term effects of Lumacaftor/Ivacaftor (Orkambi™) on exertional symptoms, exercise performance, and ventilatory responses in adults with cystic fibrosis.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Drug Combinations; Exercise Test; Female; Forced Expiratory Volume; Humans; Male; Physical Exertion; Pulmonary Ventilation; Quinolones; Treatment Outcome; Young Adult | 2020 |
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Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cystic Fibrosis; Double-Blind Method; Exercise Test; Exercise Tolerance; Female; Humans; Male; Oxygen Consumption; Quinolones | 2021 |
172 other study(ies) available for alpha-aminopyridine and lumacaftor
| Article | Year |
|---|---|
Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809.
Topics: Aminopyridines; Benzodioxoles; Bronchi; Cell Line; Cells, Cultured; Chemistry, Pharmaceutical; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Design; Drug Evaluation, Preclinical; Epithelial Cells; Homozygote; Humans; In Vitro Techniques; Lung; Models, Genetic; Mutation | 2011 |
Synthesis and evaluation of airway targeted PLGA nanoparticles for drug delivery in obstructive lung diseases.
Topics: Aminopyridines; Benzodioxoles; Drug Delivery Systems; Epithelial Cells; Humans; Lactic Acid; Lung Diseases, Obstructive; Nanoparticles; Polyglycolic Acid; Polylactic Acid-Polyglycolic Acid Copolymer; Transfection | 2012 |
Correctors of ΔF508 CFTR restore global conformational maturation without thermally stabilizing the mutant protein.
Topics: Aminopyridines; Benzodioxoles; Binding Sites; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Models, Molecular; Mutagenesis, Site-Directed; Mutant Proteins; Protein Conformation; Protein Interaction Domains and Motifs; Protein Stability; Recombinant Proteins; Sequence Deletion; Temperature | 2013 |
Pulmonology: CFTR modulators for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Lung; Mutation; Oxadiazoles; Quinolones | 2013 |
Functional rescue of a kidney anion exchanger 1 trafficking mutant in renal epithelial cells.
Topics: Aminopyridines; Animals; Anion Exchange Protein 1, Erythrocyte; Benzodioxoles; Dimethyl Sulfoxide; Dogs; Epithelial Cells; HEK293 Cells; Humans; Kidney; Madin Darby Canine Kidney Cells; Mutant Proteins; Mutation; Protein Biosynthesis; Protein Transport; Temperature | 2013 |
FDA moves on breakthrough therapies.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Drug Approval; Neoplasms; Quinolones; Terminal Care; United States | 2013 |
Mechanism-based corrector combination restores ΔF508-CFTR folding and function.
Topics: Aminopyridines; Animals; Benzodioxoles; Binding Sites; Bronchi; Cell Membrane; Cricetinae; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Endoplasmic Reticulum; Epithelial Cells; Glycosylation; Humans; Mutation; Nucleotides; Protein Folding; Protein Structure, Tertiary; Recombinant Proteins | 2013 |
Corrector VX-809 stabilizes the first transmembrane domain of CFTR.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis Transmembrane Conductance Regulator; Membrane Proteins; Mutation | 2013 |
Revertants, low temperature, and correctors reveal the mechanism of F508del-CFTR rescue by VX-809 and suggest multiple agents for full correction.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Synergism; Endoplasmic Reticulum; Humans; Kinetics; Models, Molecular; Nucleotides; Protein Folding; Protein Structure, Tertiary; Sequence Deletion; Temperature | 2013 |
VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation, Missense; Protein Conformation; Protein Folding; Protein Structure, Tertiary; Signal Transduction | 2013 |
The cystic fibrosis V232D mutation inhibits CFTR maturation by disrupting a hydrophobic pocket rather than formation of aberrant interhelical hydrogen bonds.
Topics: Amino Acid Substitution; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; HEK293 Cells; Humans; Hydrogen Bonding; Hydrophobic and Hydrophilic Interactions; Mutation; Protein Folding; Protein Modification, Translational; Protein Structure, Secondary | 2014 |
A new era in the treatment of cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genetic Therapy; Humans; Molecular Targeted Therapy; Mutation; Oxadiazoles; Quinolones | 2014 |
Genetic, cell biological, and clinical interrogation of the CFTR mutation c.3700 A>G (p.Ile1234Val) informs strategies for future medical intervention.
Topics: Adolescent; Adult; Aminopyridines; Animals; Benzodioxoles; Cell Line; Cricetinae; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Exons; HEK293 Cells; Homozygote; Humans; Isoleucine; Male; Mutation, Missense; Qatar; RNA Splicing; Valine | 2014 |
VX-809 and related corrector compounds exhibit secondary activity stabilizing active F508del-CFTR after its partial rescue to the cell surface.
Topics: Aminopyridines; Animals; Benzodioxoles; Cell Membrane; Cells, Cultured; Cricetinae; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Dose-Response Relationship, Drug; HEK293 Cells; Humans; Protein Stability; Structure-Activity Relationship; Surface Properties | 2014 |
Synergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy.
Topics: Aminopyridines; Animals; Benzodioxoles; Biological Transport; Bronchi; Cell Line; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Dogs; Drug Synergism; Epithelial Cells; Horseradish Peroxidase; Humans; Lung; Madin Darby Canine Kidney Cells; Mutation; Permeability; Protein Folding; Protein Structure, Tertiary; Respiratory Mucosa; Small Molecule Libraries; Structure-Activity Relationship | 2014 |
Direct interaction of a CFTR potentiator and a CFTR corrector with phospholipid bilayers.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis Transmembrane Conductance Regulator; Lipid Bilayers; Phospholipids; Protein Binding; Quinolones; Temperature; Unilamellar Liposomes | 2014 |
Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels.
Topics: Adenosine Triphosphate; Aminophenols; Aminopyridines; Benzodioxoles; Carrier Proteins; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; In Vitro Techniques; Intracellular Signaling Peptides and Proteins; Patch-Clamp Techniques; Peptide Fragments; Quinolones | 2014 |
Stabilizing rescued surface-localized δf508 CFTR by potentiation of its interaction with Na(+)/H(+) exchanger regulatory factor 1.
Topics: Aminopyridines; Animals; Benzodioxoles; Cell Line; Cell Membrane; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Male; Mice, Inbred C57BL; Mutation; Phosphoproteins; Protein Conformation; Protein Stability; Sodium-Hydrogen Exchangers | 2014 |
Searching for combinations of small-molecule correctors to restore f508del-cystic fibrosis transmembrane conductance regulator function and processing.
Topics: 1-Deoxynojirimycin; Aminopyridines; Benzamides; Benzodioxoles; Cell Survival; Colforsin; Cystic Fibrosis Transmembrane Conductance Regulator; Dose-Response Relationship, Drug; HeLa Cells; Humans; Thiazoles | 2014 |
The stony road to phe508del CFTR pharmacotherapy: smoothing the first rock.
Topics: Aminophenols; Aminopyridines; Base Sequence; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Male; Quinolones; Sequence Deletion | 2014 |
Function, pharmacological correction and maturation of new Indian CFTR gene mutations.
Topics: Aminopyridines; Benzodioxoles; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Gene Expression Regulation; Humans; India; Infertility, Male; Male; Male Urogenital Diseases; Mutation, Missense; Rare Diseases; Sampling Studies; Sexual Maturation; Vas Deferens | 2015 |
A cocktail drug therapy for patients with cystic fibrosis?
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Quinolones | 2014 |
Functional and pharmacological induced structural changes of the cystic fibrosis transmembrane conductance regulator in the membrane solved using SAXS.
Topics: Algorithms; Aminopyridines; Animals; Benzodioxoles; Blotting, Western; Cell Membrane; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Intracellular Membranes; Mice; Microscopy, Electron; Microsomes; Models, Molecular; Mutant Proteins; Mutation; NIH 3T3 Cells; Phosphorylation; Protein Conformation; Scattering, Small Angle; Transfection; X-Ray Diffraction | 2015 |
Remarkable progress toward new treatments for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Clinical Trials, Phase III as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Models, Animal; Forced Expiratory Volume; Humans; Mutation; Quinolones | 2014 |
SERCA and PMCA pumps contribute to the deregulation of Ca2+ homeostasis in human CF epithelial cells.
Topics: Adenosine Triphosphate; Aminopyridines; Benzodioxoles; Bronchi; Calcium; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Endoplasmic Reticulum; Epithelial Cells; Homeostasis; Humans; Mitochondria; Plasma Membrane Calcium-Transporting ATPases; Protein Binding; Sarcoplasmic Reticulum Calcium-Transporting ATPases | 2015 |
Drug combination that corrects deficient protein in cystic fibrosis improves lung function.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Humans; Peptide Fragments; Quinolones; Randomized Controlled Trials as Topic; Respiratory Function Tests | 2015 |
A molecular switch in the scaffold NHERF1 enables misfolded CFTR to evade the peripheral quality control checkpoint.
Topics: Aminopyridines; Analysis of Variance; Benzodioxoles; Biotinylation; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Cytoskeletal Proteins; Fluorescent Antibody Technique; Humans; Immunoblotting; Immunoprecipitation; Microscopy, Confocal; Phosphoproteins; Protein Conformation; Protein Folding; Protein Transport; rac1 GTP-Binding Protein; Sequence Deletion; Sodium-Hydrogen Exchangers; Temperature; Ubiquitin-Protein Ligases | 2015 |
Pseudomonas aeruginosa Reduces VX-809 Stimulated F508del-CFTR Chloride Secretion by Airway Epithelial Cells.
Topics: Aminopyridines; Benzodioxoles; Bronchi; Cell Line; Cell Membrane; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; DNA-Binding Proteins; Epithelial Cells; Humans; Mutation; Nuclear Proteins; Pseudomonas aeruginosa; Pseudomonas Infections; Transcription Factors | 2015 |
2015 American Thoracic Society International Conference.
Topics: Aminophenols; Aminopyridines; Antibodies, Monoclonal; Benzodioxoles; Congresses as Topic; Drug Therapy, Combination; Epidemiologic Methods; Gene Expression; Humans; Oxygen; Quinolones; Receptors, Interleukin-1 Type I; Respiratory System Agents; Respiratory Tract Diseases; Societies, Medical; Thoracic Surgery; United States | 2015 |
Lumacaftor/ivacaftor for patients homozygous for Phe508del-CFTR: should we curb our enthusiasm?
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Gene Deletion; Homozygote; Humans; Molecular Targeted Therapy; Quinolones | 2015 |
Radical new treatments for cystic fibrosis.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Humans; Quinolones; Randomized Controlled Trials as Topic | 2015 |
Rescuing Trafficking Mutants of the ATP-binding Cassette Protein, ABCA4, with Small Molecule Correctors as a Treatment for Stargardt Eye Disease.
Topics: Amino Acid Sequence; Aminopyridines; Anilides; ATP-Binding Cassette Transporters; Benzodioxoles; Cystic Fibrosis Transmembrane Conductance Regulator; Enzyme Inhibitors; Gene Expression; HEK293 Cells; Histone Deacetylase 6; Histone Deacetylases; HSP27 Heat-Shock Proteins; Humans; Hydroxamic Acids; Macrolides; Macular Degeneration; Molecular Sequence Data; Mutation; Protective Agents; Protein Transport; Proteolysis; Sequence Homology, Amino Acid; Signal Transduction; Stargardt Disease; Transgenes | 2015 |
Measurements of Functional Responses in Human Primary Lung Cells as a Basis for Personalized Therapy for Cystic Fibrosis.
Topics: Amino Acid Substitution; Aminopyridines; Benzodioxoles; Cells, Cultured; Clinical Trials, Phase III as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Lung; Male; Mutation, Missense; Precision Medicine | 2015 |
[Cystic Fibrosis: toward a genetic treatment; Clostridium difficile versus Clostridium difficile].
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Clostridioides difficile; Clostridium Infections; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; Humans; Mutation; Quinolones; Spores, Bacterial; Treatment Outcome | 2015 |
Capturing the Direct Binding of CFTR Correctors to CFTR by Using Click Chemistry.
Topics: Aminopyridines; Benzodioxoles; Click Chemistry; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Discovery; HEK293 Cells; Humans; Mutation; Protein Binding | 2015 |
Potentiators of Defective ΔF508-CFTR Gating that Do Not Interfere with Corrector Action.
Topics: Aminopyridines; Animals; Benzodioxoles; Cell Line; Cell Membrane; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Humans; Ion Channel Gating; Rats; Rats, Inbred F344; Structure-Activity Relationship | 2015 |
A synonymous codon change alters the drug sensitivity of ΔF508 cystic fibrosis transmembrane conductance regulator.
Topics: Action Potentials; Aminopyridines; Benzodioxoles; Codon; Cystic Fibrosis Transmembrane Conductance Regulator; HEK293 Cells; Humans; Point Mutation; Protein Binding; Protein Stability; Ubiquitination | 2016 |
Simple image-based no-wash method for quantitative detection of surface expressed CFTR.
Topics: Aminopyridines; Benzodioxoles; Cell Membrane; Cystic Fibrosis Transmembrane Conductance Regulator; Fluorescent Dyes; Gene Expression; HEK293 Cells; High-Throughput Screening Assays; Humans; Molecular Imaging; Mutation; Plasmids; Protein Engineering; Recombinant Fusion Proteins; Rosaniline Dyes; Single-Chain Antibodies; Small Molecule Libraries; Transfection | 2016 |
Low free drug concentration prevents inhibition of F508del CFTR functional expression by the potentiator VX-770 (ivacaftor).
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Bronchi; Cell Line; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Interactions; Epithelial Cells; Humans; Mutation; Quinolones | 2016 |
CFTR-regulated MAPK/NF-κB signaling in pulmonary inflammation in thermal inhalation injury.
Topics: Aminopyridines; Animals; Benzodioxoles; Cell Line; Curcumin; Cyclooxygenase 2; Cystic Fibrosis Transmembrane Conductance Regulator; Dinoprostone; Enzyme-Linked Immunosorbent Assay; Extracellular Signal-Regulated MAP Kinases; Hot Temperature; Inflammation; Inhalation; Interleukin-8; JNK Mitogen-Activated Protein Kinases; Lung Diseases; Male; Microscopy, Fluorescence; Mitogen-Activated Protein Kinases; NF-kappa B; Rats; Rats, Sprague-Dawley; Real-Time Polymerase Chain Reaction; RNA Interference; RNA, Messenger; RNA, Small Interfering; Signal Transduction; Up-Regulation | 2015 |
Breathing easier with combinations.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biomedical Research; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Industry; Drug Therapy, Combination; Humans; Mutation; Quinolones | 2015 |
Lumacaftor alone and combined with ivacaftor: preclinical and clinical trial experience of F508del CFTR correction.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Quinolones; Randomized Controlled Trials as Topic | 2016 |
Pharmacological rescue of mutant CFTR protein improves the viscoelastic properties of CF mucus.
Topics: Amiloride; Aminopyridines; Benzodioxoles; Biological Availability; Cell Culture Techniques; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Sodium Channel Blockers; Humans; Microfluidics; Models, Theoretical; Mucus; Mutant Proteins; Respiratory Mucosa | 2016 |
Phenylhydrazones as Correctors of a Mutant Cystic Fibrosis Transmembrane Conductance Regulator.
Topics: Aminopyridines; Benzodioxoles; Cell Line; Cystic Fibrosis Transmembrane Conductance Regulator; Furans; Humans; Hydrazones; Mutation; Structure-Activity Relationship; Thiophenes | 2016 |
Potentiation of ΔF508- and G551D-CFTR-Mediated Cl- Current by Novel Hydroxypyrazolines.
Topics: Aminopyridines; Animals; Bacterial Proteins; Benzodioxoles; Cell Line; Cell Line, Tumor; Cell Membrane; Cell Proliferation; Chlorides; Cyclic AMP; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Gene Deletion; Genistein; Humans; Luminescent Proteins; Mutation; Nose; Patch-Clamp Techniques; Phenylalanine; Pyrazoles; Rats; Structure-Activity Relationship; Sulfonamides | 2016 |
[Combined administration of lumacaftor and ivacaftor as a causal therapeutic approach].
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Drug Combinations; Humans; Quinolones; Randomized Controlled Trials as Topic | 2016 |
Optimal correction of distinct CFTR folding mutants in rectal cystic fibrosis organoids.
Topics: Aminopyridines; Benzodioxoles; Biopsy; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genotype; Homozygote; Humans; Mutation; Organoids; Protein Folding; Protein Transport; Rectum; Thiazoles; Treatment Outcome | 2016 |
Characterization of mitochondrial function in cells with impaired cystic fibrosis transmembrane conductance regulator (CFTR) function.
Topics: Aminopyridines; Benzodioxoles; Cells, Cultured; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Energy Metabolism; Furocoumarins; Humans; Mitochondrial Diseases; Mutation; Respiratory System | 2016 |
Ribosomal Stalk Protein Silencing Partially Corrects the ΔF508-CFTR Functional Expression Defect.
Topics: Aminopyridines; ATP-Binding Cassette Transporters; Benzodioxoles; Bronchi; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Gene Knockdown Techniques; Gene Silencing; High-Throughput Screening Assays; Humans; Peptide Elongation Factor 2; Protein Folding; Protein Stability; Ribosomal Proteins; RNA, Small Interfering; Saccharomyces cerevisiae Proteins; Yeasts | 2016 |
Mechanistic Approaches to Improve Correction of the Most Common Disease-Causing Mutation in Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Benzamides; Benzodioxoles; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Endoplasmic Reticulum; Gene Expression Regulation; Half-Life; HEK293 Cells; Humans; Mutation; Quinolones; Thiazoles | 2016 |
Cystic fibrosis drug is not cost effective, says NICE.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cost-Benefit Analysis; Cystic Fibrosis; Humans; Quinolones; Respiratory System Agents | 2016 |
Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biological Assay; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genotype; Humans; In Vitro Techniques; Mutation; Organoids; Quinolones | 2016 |
Restoration of R117H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Evaluation, Preclinical; Humans; Mutation, Missense; Protein Folding; Quinolones; Sequence Deletion | 2016 |
New Drugs 2016, part 3.
Topics: Adenosine Monophosphate; Aminopyridines; Anti-Asthmatic Agents; Antibodies, Monoclonal, Humanized; Anticoagulants; Antiemetics; Benzodioxoles; Cholagogues and Choleretics; Cystic Fibrosis Transmembrane Conductance Regulator; Deoxycholic Acid; Diarrhea; Drug Approval; Imidazoles; Irritable Bowel Syndrome; Spiro Compounds | 2016 |
The investigational Cystic Fibrosis drug Trimethylangelicin directly modulates CFTR by stabilizing the first membrane-spanning domain.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Furocoumarins; HEK293 Cells; Humans; Molecular Structure; Protein Domains | 2016 |
An "Unlikely" Pair: The Antimicrobial Synergy of Polymyxin B in Combination with the Cystic Fibrosis Transmembrane Conductance Regulator Drugs KALYDECO and ORKAMBI.
Topics: Aminophenols; Aminopyridines; Anti-Bacterial Agents; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Synergism; Drug Therapy, Combination; Humans; Polymyxin B; Pseudomonas aeruginosa; Quinolones | 2016 |
Development of HPLC and LC-MS/MS methods for the analysis of ivacaftor, its major metabolites and lumacaftor in plasma and sputum of cystic fibrosis patients treated with ORKAMBI or KALYDECO.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chromatography, High Pressure Liquid; Cystic Fibrosis; Humans; Limit of Detection; Quinolones; Sputum; Tandem Mass Spectrometry | 2016 |
Direct Measurement of Trafficking of the Cystic Fibrosis Transmembrane Conductance Regulator to the Cell Surface and Binding to a Chemical Chaperone.
Topics: Aminopyridines; Benzodioxoles; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; HEK293 Cells; Humans; Hydrogen-Ion Concentration; Interferometry; Microscopy, Fluorescence; Mutation; Protein Binding; Protein Transport; Reproducibility of Results; Single Molecule Imaging; Temperature; Transport Vesicles | 2017 |
Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Cell Line; Cell Membrane; Cell-Free System; Chromatography; Cricetinae; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Hot Temperature; Humans; Mutation; Patch-Clamp Techniques; Protein Denaturation; Quinolones | 2017 |
Pregnancy among cystic fibrosis women in the era of CFTR modulators.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Pregnancy; Pregnancy Complications; Pregnancy Outcome; Pregnancy Rate; Pregnancy, High-Risk; Quinolones; Risk Adjustment; United States | 2017 |
Effects of Lumacaftor/Ivacaftor in a Pediatric Cohort Homozygous for F508del-CFTR.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Quinolones | 2017 |
Corrector VX-809 promotes interactions between cytoplasmic loop one and the first nucleotide-binding domain of CFTR.
Topics: Amino Acid Sequence; Aminopyridines; Benzodioxoles; Binding Sites; Cystic Fibrosis Transmembrane Conductance Regulator; Cytoplasm; HEK293 Cells; Humans; Protein Binding | 2017 |
Orkambi in patients with severe disease - Bumps in the road to CFTR modulation.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Quinolones | 2017 |
Direct Binding of the Corrector VX-809 to Human CFTR NBD1: Evidence of an Allosteric Coupling between the Binding Site and the NBD1:CL4 Interface.
Topics: Allosteric Regulation; Aminopyridines; Benzodioxoles; Binding Sites; Carrier Proteins; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Intracellular Signaling Peptides and Proteins; Protein Binding; Protein Structure, Secondary; Protein Structure, Tertiary | 2017 |
Cell Class-Dependent Intracortical Connectivity and Output Dynamics of Layer 6 Projection Neurons of the Rat Primary Visual Cortex.
Topics: Action Potentials; Aminopyridines; Animals; Benzodioxoles; Biophysics; Brain Mapping; Electric Stimulation; Functional Laterality; Geniculate Bodies; Glucose Transporter Type 2; In Vitro Techniques; Lysine; Male; Nerve Net; Neurons; Patch-Clamp Techniques; Rats; Rats, Wistar; Synapses; Visual Cortex; Visual Pathways | 2018 |
Lumacaftor/ivacaftor combination therapy for cystic fibrosis: A nationwide survey among clinicians.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Progression; Drug Combinations; Drug Therapy, Combination; Forced Expiratory Volume; Homozygote; Humans; Italy; Mutation; Quinolones; Surveys and Questionnaires; Treatment Outcome | 2018 |
Src kinase inhibition reduces inflammatory and cytoskeletal changes in ΔF508 human cholangiocytes and improves cystic fibrosis transmembrane conductance regulator correctors efficacy.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Biliary Tract; Cell Culture Techniques; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Cytokines; Cytoskeleton; Epithelial Cells; Fluorescent Antibody Technique; Humans; Induced Pluripotent Stem Cells; Inflammation; Mice; Microscopy, Confocal; Pyrimidines; Quinolones; Signal Transduction; src-Family Kinases | 2018 |
Vertex CF data wow Wall Street.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Drug Combinations; Humans; Quinolones | 2017 |
Cigarette smoke activates CFTR through ROS-stimulated cAMP signaling in human bronchial epithelial cells.
Topics: Aminophenols; Aminopyridines; Autocrine Communication; Benzodioxoles; Bronchi; Calcium Signaling; Cell Line; Cyclic AMP; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Humans; Mutation; Oxidative Stress; Quinolones; Reactive Oxygen Species; Receptors, Prostaglandin E, EP4 Subtype; Second Messenger Systems; Secretory Pathway; Tobacco Smoke Pollution | 2018 |
Identification of a targeted and testable antiarrhythmic therapy for long-QT syndrome type 2 using a patient-specific cellular model.
Topics: Action Potentials; Adolescent; Adult; Aminopyridines; Anti-Arrhythmia Agents; Benzodioxoles; Calcium; ERG1 Potassium Channel; Female; Humans; Induced Pluripotent Stem Cells; Long QT Syndrome; Male; Middle Aged; Myocytes, Cardiac; Protein Transport; Treatment Outcome | 2018 |
Advancing precision medicine for the treatment of long-QT syndrome type 2: shedding light on lumacaftor.
Topics: Aminopyridines; Benzodioxoles; Humans; Long QT Syndrome; Precision Medicine | 2018 |
Lumacaftor (VX-809) restores the ability of CF macrophages to phagocytose and kill Pseudomonas aeruginosa.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Forced Expiratory Volume; Humans; Macrophages; Mutation; Phagocytosis; Pseudomonas aeruginosa; Pseudomonas Infections; Quinolones | 2018 |
Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Case-Control Studies; Chromatography, Liquid; Cystic Fibrosis; High-Throughput Screening Assays; Humans; Quinolones; Tandem Mass Spectrometry | 2017 |
Quorum Sensing Down-Regulation Counteracts the Negative Impact of
Topics: Aminopyridines; Bacterial Proteins; Benzodioxoles; Cell Line; Cells, Cultured; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Down-Regulation; Epithelial Cells; Gene Expression Regulation, Bacterial; Humans; Infant; Mutation; Piperazines; Pseudomonas aeruginosa; Pseudomonas Infections; Quinazolines; Quorum Sensing; Respiratory System; Trans-Activators | 2017 |
Nasospheroids permit measurements of CFTR-dependent fluid transport.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biological Transport; Colforsin; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Mutation; Nasal Mucosa; Particle Size; Precision Medicine; Quinolones; Spheroids, Cellular | 2017 |
Complexity of phenotypes induced by p.Asn1303Lys-CFTR correlates with difficulty to rescue and activate this protein.
Topics: Aminopyridines; Benzodioxoles; Blotting, Western; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; HeLa Cells; Humans; Leupeptins; Mutation | 2017 |
Cystic Fibrosis Transmembrane Conductance Regulator Attaches Tumor Suppressor PTEN to the Membrane and Promotes Anti Pseudomonas aeruginosa Immunity.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Gene Expression Regulation; Humans; Mice; Mice, Inbred C57BL; Mice, Knockout; Models, Molecular; Monocytes; Phosphatidylinositol 3-Kinases; Protein Binding; Protein Conformation; Protein Transport; Proto-Oncogene Proteins c-akt; Pseudomonas aeruginosa; Pseudomonas Infections; PTEN Phosphohydrolase; Quinolones; Signal Transduction | 2017 |
Synthesis and biological evaluation of novel thiazole- VX-809 hybrid derivatives as F508del correctors by QSAR-based filtering tools.
Topics: Aminopyridines; Benzodioxoles; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation; Quantitative Structure-Activity Relationship; Thiazoles | 2018 |
Vx-809/Vx-770 treatment reduces inflammatory response to Pseudomonas aeruginosa in primary differentiated cystic fibrosis bronchial epithelial cells.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Bronchi; Cells, Cultured; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Humans; Interleukin-8; Mutation; Pseudomonas aeruginosa; Pseudomonas Infections; Quinolones | 2018 |
High-Content Surface and Total Expression siRNA Kinase Library Screen with VX-809 Treatment Reveals Kinase Targets that Enhance F508del-CFTR Rescue.
Topics: Aminopyridines; Benzodioxoles; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Evaluation, Preclinical; Drug Synergism; Drug Therapy, Combination; Flow Cytometry; Fluorescent Dyes; Gene Knockdown Techniques; HEK293 Cells; High-Throughput Screening Assays; Humans; Mutation; Phosphotransferases; Protein Kinase Inhibitors; RNA, Small Interfering; Treatment Outcome | 2018 |
Biomarkers: Their Role in CFTR Modulator Therapies from Early Development to the Clinic.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Quinolones | 2018 |
Guanosine monophosphate reductase 1 is a potential therapeutic target for Alzheimer's disease.
Topics: Adaptor Proteins, Signal Transducing; Alzheimer Disease; Aminopyridines; Amyloid beta-Peptides; Animals; Benzodioxoles; C-Reactive Protein; Databases, Genetic; Disease Models, Animal; Enzyme Inhibitors; Glycogen Synthase Kinase 3 beta; GMP Reductase; Mice; Molecular Targeted Therapy; Nerve Tissue Proteins; Phosphorylation; Proto-Oncogene Proteins c-met; Repressor Proteins; tau Proteins; Wnt Signaling Pathway | 2018 |
Chronic β2AR stimulation limits CFTR activation in human airway epithelia.
Topics: Adrenergic beta-2 Receptor Agonists; Albuterol; Aminophenols; Aminopyridines; Benzodioxoles; Cell Line; Cilia; Cyclic AMP; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Interactions; Epithelial Cells; Humans; Mutation; Quinolones; Respiratory Mucosa; Time Factors | 2018 |
Personalised CFTR pharmacotherapeutic response testing and therapy of cystic fibrosis.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Male; Mutation; Precision Medicine; Quinolones | 2018 |
Comprehensive mapping of cystic fibrosis mutations to CFTR protein identifies mutation clusters and molecular docking predicts corrector binding site.
Topics: Aminopyridines; Benzodioxoles; Binding Sites; Cluster Analysis; Cystic Fibrosis Transmembrane Conductance Regulator; Databases, Protein; HEK293 Cells; Humans; Indoles; Molecular Docking Simulation; Mutation; Protein Binding; Protein Folding; Protein Structure, Tertiary | 2018 |
Correctors of the Major Cystic Fibrosis Mutant Interact through Membrane-Spanning Domains.
Topics: Aminopyridines; Benzamides; Benzodioxoles; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; HEK293 Cells; Humans; Mutation; Phenylalanine; Protein Structure, Tertiary; Thiazoles | 2018 |
A potential strategy for reducing cysts in autosomal dominant polycystic kidney disease with a CFTR corrector.
Topics: Aminopyridines; Animals; Benzodioxoles; Calcium; Cell Line; Cell Proliferation; Cyclic AMP; Cystic Fibrosis Transmembrane Conductance Regulator; Cysts; Heat-Shock Proteins; Kidney; Mice; Mice, Inbred C57BL; Polycystic Kidney, Autosomal Dominant; Transcription Factor CHOP | 2018 |
Lipophilicity of the Cystic Fibrosis Drug, Ivacaftor (VX-770), and Its Destabilizing Effect on the Major CF-causing Mutation: F508del.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Freeze Drying; HEK293 Cells; Humans; Microscopy, Fluorescence; Mutation; Protein Stability; Quinolones; Sulfate Transporters | 2018 |
Rescue of CFTR NBD2 mutants N1303K and S1235R is influenced by the functioning of the autophagosome.
Topics: Aminopyridines; Animals; Autophagosomes; Autophagy; Benzodioxoles; Biological Transport; Blotting, Western; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Electric Conductivity; Leucine; Mutant Proteins; Mutation; Small Molecule Libraries | 2018 |
F508del-CFTR is not corrected by thymosin α1.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Homozygote; Humans; Mutation; Thymalfasin | 2018 |
Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulators.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Therapy, Combination; HEK293 Cells; Humans; Mutation; Quinolones | 2018 |
A novel triple combination of pharmacological chaperones improves F508del-CFTR correction.
Topics: Aminopyridines; Animals; Benzodioxoles; Cell Line; Cell Polarity; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Synergism; Drug Therapy, Combination; Epithelial Cells; Humans; Membrane Potentials; Mutation; Protein Domains | 2018 |
Prolonged co-treatment with HGF sustains epithelial integrity and improves pharmacological rescue of Phe508del-CFTR.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cell Line; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Hepatocyte Growth Factor; Humans; Models, Biological; Mutant Proteins; Quinolones; Treatment Outcome | 2018 |
Cost-effectiveness analysis of lumacaftor and ivacaftor combination for the treatment of patients with cystic fibrosis in the United States.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Cost-Benefit Analysis; Cystic Fibrosis; Female; Humans; Male; Quality-Adjusted Life Years; Quinolones; United States | 2018 |
The cystic fibrosis airway milieu enhances rescue of F508del in a pre-clinical model.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Bronchi; Cell Line; Colforsin; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Humans; Inflammation; Mutation; Quinolones; Respiratory System | 2018 |
Cystic Fibrosis: Emerging Understanding and Therapies.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chronic Disease; Combined Modality Therapy; Comprehension; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Management; Disease Progression; Drug Approval; Female; Genetic Predisposition to Disease; Humans; Lung Transplantation; Male; Prognosis; Quinolones; Risk Assessment; Severity of Illness Index; Survival Analysis; United States; United States Food and Drug Administration | 2019 |
Might Brushed Nasal Cells Be a Surrogate for CFTR Modulator Clinical Response?
Topics: Aminopyridines; Benzodioxoles; Biomarkers; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Nasal Cavity; Nasal Mucosa; Treatment Outcome | 2019 |
Influenza-mediated reduction of lung epithelial ion channel activity leads to dysregulated pulmonary fluid homeostasis.
Topics: Aminopyridines; Animals; Benzodioxoles; Bronchi; Cells, Cultured; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Models, Animal; Dogs; Epithelial Cells; Epithelial Sodium Channels; Female; Humans; Influenza A virus; Influenza, Human; Madin Darby Canine Kidney Cells; Male; Mice; Primary Cell Culture; Pulmonary Edema; Respiratory Distress Syndrome; Respiratory Mucosa; Sodium-Potassium-Exchanging ATPase; Water-Electrolyte Balance | 2018 |
Structural mechanisms for defective CFTR gating caused by the Q1412X mutation, a severe Class VI pathogenic mutation in cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Chloride Channel Agonists; CHO Cells; Cricetulus; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Ion Channel Gating; Mutation; Protein Conformation; Quinolones | 2019 |
Compassionate Use of Lumacaftor/Ivacaftor in Cystic Fibrosis: Spanish Experience.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Compassionate Use Trials; Cystic Fibrosis; Drug Combinations; Female; Humans; Male; Middle Aged; Quinolones; Retrospective Studies; Spain; Young Adult | 2018 |
Folding-function relationship of the most common cystic fibrosis-causing CFTR conductance mutants.
Topics: Alleles; Aminophenols; Aminopyridines; Benzodioxoles; Biopsy; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Genotype; HEK293 Cells; Humans; Mutation; Organoids; Protein Folding; Protein Structure, Tertiary; Quinolones; Rectum; Transfection | 2019 |
Inhalation treatment of cystic fibrosis with lumacaftor and ivacaftor co-delivered by nanostructured lipid carriers.
Topics: Administration, Inhalation; Aminophenols; Aminopyridines; Animals; Benzodioxoles; Cell Line; Chloride Channel Agonists; Cystic Fibrosis; Drug Carriers; Drug Combinations; Humans; Lipids; Lung; Mice, Transgenic; Nanostructures; Quinolones | 2019 |
The study of CFTR modulators in the very young.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Child, Preschool; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Quinolones | 2019 |
Extent of rescue of F508del-CFTR function by VX-809 and VX-770 in human nasal epithelial cells correlates with SNP rs7512462 in SLC26A9 gene in F508del/F508del Cystic Fibrosis patients.
Topics: Alleles; Aminophenols; Aminopyridines; Antiporters; Base Sequence; Benzodioxoles; Body Mass Index; Case-Control Studies; Cellular Reprogramming; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Diffusion Chambers, Culture; Epithelial Cells; Gene Expression; Genotype; Humans; Models, Biological; Nasal Mucosa; Polymorphism, Single Nucleotide; Primary Cell Culture; Quinolones; Sequence Deletion; Sulfate Transporters; Sweat | 2019 |
Lumacaftor-rescued F508del-CFTR has a modified bicarbonate permeability.
Topics: Aminopyridines; Anion Transport Proteins; Benzodioxoles; Bicarbonates; Cell Membrane Permeability; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Ion Transport; Membrane Transport Modulators; Mutation | 2019 |
HDAC inhibitors rescue multiple disease-causing CFTR variants.
Topics: Aminopyridines; Benzodioxoles; Cell Membrane; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Histone Deacetylase Inhibitors; Humans; Hydroxamic Acids; Mutation; Panobinostat; Protein Transport; Sequence Deletion; Sulfonamides | 2019 |
Differential thermostability and response to cystic fibrosis transmembrane conductance regulator potentiators of human and mouse F508del-CFTR.
Topics: Adenosine Triphosphate; Aminophenols; Aminopyridines; Animals; Base Sequence; Benzodioxoles; Chloride Channel Agonists; CHO Cells; Colforsin; Cricetulus; Cyclic AMP-Dependent Protein Kinases; Cystic Fibrosis Transmembrane Conductance Regulator; Gene Expression; Genistein; Ion Transport; Mice; NIH 3T3 Cells; Patch-Clamp Techniques; Protein Stability; Quinolones; Sequence Deletion; Species Specificity; Temperature; Transgenes | 2019 |
Phenotyping ciliary dynamics and coordination in response to CFTR-modulators in Cystic Fibrosis respiratory epithelial cells.
Topics: Algorithms; Aminophenols; Aminopyridines; Benzodioxoles; Bronchi; Cilia; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Progression; Epithelial Cells; Genotype; Humans; Microscopy; Oscillometry; Phenotype; Quinolones; Video Recording | 2019 |
Unexpected analytical interference in isavuconazole UV determination in a child in therapy with lumacaftor/ivacaftor for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Antifungal Agents; Antineoplastic Combined Chemotherapy Protocols; Benzodioxoles; Cystic Fibrosis; Female; Humans; Male; Nitriles; Pyridines; Quinolones; Triazoles | 2019 |
Negotiations between the NHS and Vertex on access to lumacaftor and ivacaftor become protracted.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Drug Combinations; Drug Industry; England; Humans; Negotiating; Quinolones; State Medicine | 2019 |
The CFTR Corrector, VX-809 (Lumacaftor), Rescues ABCA4 Trafficking Mutants: a Potential Treatment for Stargardt Disease.
Topics: Aminopyridines; Anilides; ATP-Binding Cassette Transporters; Benzodioxoles; Cell Membrane; Cystic Fibrosis Transmembrane Conductance Regulator; Gene Expression Regulation; HEK293 Cells; HSP27 Heat-Shock Proteins; Humans; Hydroxamic Acids; Leupeptins; Lysosomes; Macular Degeneration; Mutation; Protein Transport; Stargardt Disease | 2019 |
Successful Pregnancy of a Patient with Cystic Fibrosis Genotype F508del/ F508del and Progressed Pulmonary Destruction on lumacaftor/ivacaftor.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Genotype; Humans; Lung; Mutation; Pregnancy; Pregnancy Outcome; Quinolones | 2019 |
Effect of Lumacaftor/Ivacaftor on Pulmonary Exacerbation Rates in Members with Cystic Fibrosis in a Medicaid Population.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Humans; Lung; Male; Medicaid; Middle Aged; Mutation; Quinolones; United States; Young Adult | 2019 |
Cystic fibrosis transmembrane conductance regulator modulators reduce the risk of recurrent acute pancreatitis among adult patients with pancreas sufficient cystic fibrosis.
Topics: Adult; Aged; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Exocrine Pancreatic Insufficiency; Female; Humans; Indoles; Male; Middle Aged; Quinolones; Retrospective Studies | 2019 |
Combination Therapy with Lumacaftor-Ivacaftor in Cystic Fibrosis. Keeping It Real.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Humans; Quinolones | 2020 |
Slowing ribosome velocity restores folding and function of mutant CFTR.
Topics: Aminopyridines; Animals; Benzodioxoles; Bronchi; Colon; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelium; Female; Gene Silencing; HEK293 Cells; Humans; Ileum; Indoles; Male; Mice; Mice, Inbred C57BL; Mice, Transgenic; Mutant Proteins; Mutation; Pancreas; Patch-Clamp Techniques; Protein Conformation; Protein Folding; Rats; Ribosomal Proteins; Ribosomes | 2019 |
Unravelling the Regions of Mutant F508del-CFTR More Susceptible to the Action of Four Cystic Fibrosis Correctors.
Topics: Aminopyridines; Benzamides; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Gene Expression Regulation; HEK293 Cells; Humans; Indoles; Mutant Proteins; Mutation; Quinazolines; Thiazoles | 2019 |
CFTR transmembrane segments are impaired in their conformational adaptability by a pathogenic loop mutation and dynamically stabilized by Lumacaftor.
Topics: Amino Acid Sequence; Aminophenols; Aminopyridines; Benzodioxoles; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Molecular Conformation; Mutation; Protein Folding; Structure-Activity Relationship | 2020 |
Different CFTR modulator combinations downregulate inflammation differently in cystic fibrosis.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Cytokines; Down-Regulation; Drug Therapy, Combination; Female; Humans; Indoles; Inflammation; Interleukin-18; Interleukin-1beta; Male; Monocytes; Quinolones; Tumor Necrosis Factor-alpha; Young Adult | 2020 |
Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; HEK293 Cells; Humans; Muscle Fibers, Skeletal; Mutation; Proteasome Endopeptidase Complex; Sarcoglycanopathies; Sarcoglycans | 2020 |
The dual phosphodiesterase 3/4 inhibitor RPL554 stimulates rare class III and IV CFTR mutants.
Topics: Aminopyridines; Animals; Benzodioxoles; Bronchi; Cell Line; Colforsin; Cyclic AMP; Cyclic Nucleotide Phosphodiesterases, Type 3; Cyclic Nucleotide Phosphodiesterases, Type 4; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Isoquinolines; Mutation; Phosphodiesterase 3 Inhibitors; Phosphodiesterase 4 Inhibitors; Primary Cell Culture; Pyrimidinones; Rats; Rats, Inbred F344; Thyroid Epithelial Cells; Transgenes | 2020 |
Structural Consequences of the 1,2,3-Triazole as an Amide Bioisostere in Analogues of the Cystic Fibrosis Drugs VX-809 and VX-770.
Topics: Amides; Aminophenols; Aminopyridines; Benzodioxoles; Crystallography, X-Ray; Cystic Fibrosis; Humans; Models, Molecular; Molecular Structure; Quantum Theory; Quinolones; Triazoles | 2020 |
Can lumacaftor-ivacaftor reverse glucose-tolerance abnormalities in cystic fibrosis?
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Glucose; Humans; Quinolones | 2020 |
Correctors modify the bicarbonate permeability of F508del-CFTR.
Topics: Aminopyridines; Animals; Benzodioxoles; Bicarbonates; Cell Membrane; Cell Membrane Permeability; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation; Protein Transport; Rats; Thyroid Gland | 2020 |
Comment on "Effect of one-year lumacaftor-ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients".
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Glucose; Humans; Quinolones | 2020 |
Changes in LCI in F508del/F508del patients treated with lumacaftor/ivacaftor: Results from the prospect study.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Male; Middle Aged; Mutation; Quinolones; Respiratory Function Tests; United States | 2020 |
Drug allergy to CFTR modulator therapy associated with lumacaftor-specific CD4
Topics: Aminophenols; Aminopyridines; Benzodioxoles; CD4-Positive T-Lymphocytes; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Hypersensitivity; Female; Humans; Quinolones; Young Adult | 2021 |
GM1 as Adjuvant of Innovative Therapies for Cystic Fibrosis Disease.
Topics: Adjuvants, Immunologic; Aminophenols; Aminopyridines; Benzodioxoles; Bronchi; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; G(M1) Ganglioside; Humans; Mutation; Quinolones; Therapies, Investigational | 2020 |
Characterization of the mechanism of action of RDR01752, a novel corrector of F508del-CFTR.
Topics: Aminopyridines; Benzodioxoles; Bronchi; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Discovery; Humans; Indoles; Mutation; Organoids; Protein Transport | 2020 |
Full Rescue of F508del-CFTR Processing and Function by CFTR Modulators Can Be Achieved by Removal of Two Regulatory Regions.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cell Line; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation; Protein Domains; Quinolones; Regulatory Sequences, Nucleic Acid; Signal Transduction | 2020 |
Phosphorylation of the Chaperone-Like HspB5 Rescues Trafficking and Function of F508del-CFTR.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Cell Line; Cell Membrane; Crystallins; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Heat-Shock Proteins; HEK293 Cells; Humans; Male; Mice; Molecular Chaperones; Mutation; Phenylalanine; Phosphorylation; Proteasome Endopeptidase Complex; Protein Transport; Quinolones | 2020 |
CFTR modulator therapy for cystic fibrosis caused by the rare c.3700A>G mutation.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cells, Cultured; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Indoles; Mutant Proteins; Mutation, Missense; Pyrazoles; Pyridines; Pyrrolidines; Quinolones | 2021 |
Organoids as a personalized medicine tool for ultra-rare mutations in cystic fibrosis: The case of S955P and 1717-2A>G.
Topics: Alleles; Aminophenols; Aminopyridines; Benzodioxoles; Blotting, Western; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Electrophysiology; Fluorescent Antibody Technique; Genotype; Humans; Indoles; Mutation; Precision Medicine; Quinolones | 2020 |
Reduced Intestinal Inflammation With Lumacaftor/Ivacaftor in Adolescents With Cystic Fibrosis.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Inflammation; Lung; Mutation; Quinolones | 2020 |
An integrated drug repurposing strategy for the rapid identification of potential SARS-CoV-2 viral inhibitors.
Topics: Aminopyridines; Angiotensin-Converting Enzyme 2; Benzodioxoles; Betacoronavirus; Binding Sites; Computational Biology; Coronavirus Infections; COVID-19; Drug Discovery; Drug Repositioning; Humans; Molecular Docking Simulation; Molecular Dynamics Simulation; Pandemics; Peptidyl-Dipeptidase A; Pneumonia, Viral; Protein Binding; Protein Conformation; Protein Domains; Protein Interaction Maps; SARS-CoV-2; Simeprevir; Spike Glycoprotein, Coronavirus | 2020 |
Promise and Potential Peril With Lumacaftor for the Trafficking Defective Type 2 Long-QT Syndrome-Causative Variants, p.G604S, p.N633S, and p.R685P, Using Patient-Specific Re-Engineered Cardiomyocytes.
Topics: Action Potentials; Aminopyridines; Benzodioxoles; ERG1 Potassium Channel; Humans; Induced Pluripotent Stem Cells; Long QT Syndrome; Mutation, Missense; Myocytes, Cardiac; Phenotype; Polymorphism, Single Nucleotide; Protein Subunits | 2020 |
Choice of Differentiation Media Significantly Impacts Cell Lineage and Response to CFTR Modulators in Fully Differentiated Primary Cultures of Cystic Fibrosis Human Airway Epithelial Cells.
Topics: Aminopyridines; Benzodioxoles; Cell Differentiation; Cell Lineage; Culture Media; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Diffusion Chambers, Culture; Epithelial Cells; Gene Expression Regulation; Goblet Cells; H(+)-K(+)-Exchanging ATPase; Humans; Hydrogen-Ion Concentration; Primary Cell Culture; Respiratory Mucosa; Sequence Analysis, RNA; Transcriptome | 2020 |
Discovery of novel VX-809 hybrid derivatives as F508del-CFTR correctors by molecular modeling, chemical synthesis and biological assays.
Topics: Aminopyridines; Benzodioxoles; Cell Line; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Design; Humans; Molecular Docking Simulation; Mutation; Protein Binding; Protein Domains | 2020 |
Molecular Docking and QSAR Studies as Computational Tools Exploring the Rescue Ability of F508del CFTR Correctors.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Molecular Docking Simulation; Mutation; Quantitative Structure-Activity Relationship | 2020 |
Quantitative Method for the Analysis of Ivacaftor, Hydroxymethyl Ivacaftor, Ivacaftor Carboxylate, Lumacaftor, and Tezacaftor in Plasma and Sputum Using Liquid Chromatography With Tandem Mass Spectrometry and Its Clinical Applicability.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chromatography, Liquid; Cystic Fibrosis; Drug Combinations; Humans; Indoles; Mutation; Plasma; Quinolones; Sputum; Tandem Mass Spectrometry | 2021 |
The PROSPECT Is Bright for CFTR Modulators.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Quinolones; Treatment Outcome | 2021 |
Lumacaftor/ivacaftor in cystic fibrosis: effects on glucose metabolism and insulin secretion.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Blood Glucose; Case-Control Studies; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Follow-Up Studies; Homozygote; Humans; Insulin Secretion; Male; Mutation; Prognosis; Quinolones; Retrospective Studies; Young Adult | 2021 |
Novel Correctors and Potentiators Enhance Translational Readthrough in CFTR Nonsense Mutations.
Topics: Aminophenols; Aminopyridines; Animals; Benzoates; Benzodioxoles; Benzopyrans; Cell Line; Chlorides; Codon, Nonsense; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Humans; Ion Transport; Protein Biosynthesis; Pyrans; Pyrazoles; Quinolones; Rats; Recovery of Function; Thyroid Epithelial Cells | 2021 |
Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cells, Cultured; Child, Preschool; Chloride Channel Agonists; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Female; Genotype; Humans; Mutation, Missense; Nasal Mucosa; Pancreatic Elastase; Quinolones | 2021 |
Co-Translational Folding of the First Transmembrane Domain of ABC-Transporter CFTR is Supported by Assembly with the First Cytosolic Domain.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis Transmembrane Conductance Regulator; Cytosol; Evolution, Molecular; Genes, Suppressor; HEK293 Cells; HeLa Cells; Humans; Models, Molecular; Peptide Hydrolases; Protein Biosynthesis; Protein Domains; Protein Folding; Protein Structure, Secondary | 2021 |
Multidisciplinary Approaches Identify Compounds that Bind to Human ACE2 or SARS-CoV-2 Spike Protein as Candidates to Block SARS-CoV-2-ACE2 Receptor Interactions.
Topics: Aminopyridines; Angiotensin-Converting Enzyme 2; Animals; Antiviral Agents; Benzodioxoles; Cell Line; Chlorocebus aethiops; COVID-19 Drug Treatment; Drug Evaluation, Preclinical; Drug Repositioning; Evans Blue; Humans; Molecular Docking Simulation; Phenylalanine; Protein Binding; SARS-CoV-2; Spike Glycoprotein, Coronavirus; Sulfones; Surface Plasmon Resonance; Vero Cells; Virus Attachment; Virus Replication | 2021 |
Lumacaftor and Matrine: Possible Therapeutic Combination to Counteract the Inflammatory Process in Cystic Fibrosis.
Topics: A549 Cells; Alkaloids; Aminopyridines; Benzodioxoles; Cell Death; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Synergism; Drug Therapy, Combination; Humans; Inflammation; Matrines; Models, Biological; Oxidative Stress; Quinolizidines; Quinolizines; Reactive Oxygen Species; Signal Transduction | 2021 |
Deep-learning based repurposing of FDA-approved drugs against
Topics: Aminopyridines; Antifungal Agents; Benzodioxoles; Candida albicans; Candidiasis; Cyclopropanes; Deep Learning; Drug Approval; Drug Repositioning; Folic Acid Antagonists; Humans; Lactams, Macrocyclic; Molecular Docking Simulation; Molecular Dynamics Simulation; Proline; Rifampin; Sulfonamides; Tetrahydrofolate Dehydrogenase; United States; United States Food and Drug Administration | 2022 |
Long-term safety of lumacaftor-ivacaftor in children aged 2-5 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a multicentre, phase 3, open-label, extension study.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Child, Preschool; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation; Quinolones | 2021 |
Lumacaftor-ivacaftor effects on cystic fibrosis-related liver involvement in adolescents with homozygous F508 del-CFTR.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Liver; Mutation; Prospective Studies; Quinolones | 2022 |
Restoration of exocrine pancreatic function in child with lumacaftor/ivacaftor therapy in cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Quinolones | 2022 |
VX-809 mitigates disease in a mouse model of autosomal dominant polycystic kidney disease bearing the R3277C human mutation.
Topics: Aminopyridines; Animals; Benzodioxoles; Cystic Fibrosis Transmembrane Conductance Regulator; Cysts; Female; Kidney; Male; Mice; Mice, Inbred C57BL; Polycystic Kidney, Autosomal Dominant | 2021 |
Isotope dilution LC-MS/MS quantification of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators ivacaftor, lumacaftor, tezacaftor, elexacaftor, and their major metabolites in human serum.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chromatography, Liquid; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Indoles; Isotopes; Mutation; Pyrazoles; Pyridines; Pyrrolidines; Quinolones; Tandem Mass Spectrometry | 2022 |
NBD2 Is Required for the Rescue of Mutant F508del CFTR by a Thiazole-Based Molecule: A Class II Corrector for the Multi-Drug Therapy of Cystic Fibrosis.
Topics: Aminopyridines; Benzodioxoles; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Gene Expression Regulation; HEK293 Cells; Humans; Mutant Proteins; Phenylalanine; Thiazoles | 2021 |
Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR.
Topics: Aminopyridines; Benzodioxoles; Binding Sites; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Therapy, Combination; HEK293 Cells; Humans; Mutation; Protein Domains; Protein Folding; Protein Structure, Tertiary; Pyrazoles; Pyridines; Pyrrolidines | 2021 |
Comprehensive Analysis of Combinatorial Pharmacological Treatments to Correct Nonsense Mutations in the CFTR Gene.
Topics: Aminopyridines; Benzodioxoles; Bronchi; Chloride Channel Agonists; Codon, Nonsense; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelial Cells; Humans; Nonsense Mediated mRNA Decay; Pyrazoles; Pyridines; Pyrrolidines | 2021 |
Synergy in Cystic Fibrosis Therapies: Targeting SLC26A9.
Topics: Aminophenols; Aminopyridines; Antiporters; Benzodioxoles; Bronchi; Cell Line; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Gene Expression Regulation; Gene Knockdown Techniques; HEK293 Cells; Humans; Indoles; Molecular Targeted Therapy; Mutation; Organ Culture Techniques; Pyrazoles; Pyridines; Quinolines; Sulfate Transporters; Zonula Occludens-1 Protein | 2021 |
Liquid chromatography-mass spectrometric methods for trace quantification of potential genotoxic impurities in ivacaftor and lumacaftor.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Boronic Acids; Chromatography, High Pressure Liquid; Chromatography, Liquid; DNA Damage; Nitrophenols; Quinolones; Reproducibility of Results | 2022 |
Mechanism of CFTR correction by type I folding correctors.
Topics: Aminopyridines; Animals; Benzodioxoles; Binding Sites; Cell Membrane; CHO Cells; Cricetulus; Cryoelectron Microscopy; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; HEK293 Cells; Humans; Hydrophobic and Hydrophilic Interactions; Indoles; Molecular Chaperones; Mutation; Protein Domains; Protein Folding; Sf9 Cells; Transfection | 2022 |
Radiomics-derived morphological features predict pulmonary function response during lumacaftor/ivacaftor therapy in patients with cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Drug Combinations; Humans; Quinolones; Tomography, X-Ray Computed; Unsupervised Machine Learning | 2022 |
Diversity of approaches in artificial intelligence: an opportunity for discoveries in thoracic imaging.
Topics: Aminophenols; Aminopyridines; Artificial Intelligence; Benzodioxoles; Cystic Fibrosis; Humans; Quinolones; Tomography, X-Ray Computed; Unsupervised Machine Learning | 2022 |
Changes in Glucose Breath Test in Cystic Fibrosis Patients Treated With 1 Month of Lumacaftor/Ivacaftor.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Breath Tests; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Glucose; Humans; Hydrogen; Longitudinal Studies; Male; Mutation; Quinolones; Young Adult | 2022 |
Change in exercise capacity measured by Cardio-pulmonary Exercise Testing (CPET) in Danish people with cystic fibrosis after initiation of treatment with Lumacaftor/Ivacaftor and Tezacaftor/Ivacaftor.
Topics: Aminophenols; Aminopyridines; Bacterial Toxins; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Denmark; Drug Combinations; Exercise Test; Exercise Tolerance; Humans; Indoles; Mutation; Prospective Studies; Quinolones | 2022 |
CFTR Rescue by Lumacaftor (VX-809) Induces an Extensive Reorganization of Mitochondria in the Cystic Fibrosis Bronchial Epithelium.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Epithelium; Humans; Infant, Newborn; Mitochondria; Proteome | 2022 |
T-cell-mediated hypersensitivity to lumacaftor and ivacaftor in cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Drug Combinations; Humans; Mutation; Quinolones; T-Lymphocytes | 2022 |
Ivacaftor, not ivacaftor/lumacaftor, associated with lower pulmonary inflammation in preschool cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child, Preschool; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Mutation; Pneumonia; Quinolones | 2022 |
Profiling the response to lumacaftor-ivacaftor in children with cystic between fibrosis and new insight from a French-Italian real-life cohort.
Topics: Adolescent; Aminophenols; Aminopyridines; Anti-Bacterial Agents; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Fibrosis; Forced Expiratory Volume; Humans; Mutation | 2022 |
Drug desensitization to lumacaftor/ivacaftor: A fast lane to drug tolerance.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Tolerance; Exanthema; Female; Forced Expiratory Volume; Humans; Infant; Mutation | 2023 |
Human epididymis protein 4 (HE4) plasma concentration inversely correlates with the improvement of cystic fibrosis lung disease in p.Phe508del-CFTR homozygous cases treated with the CFTR modulator lumacaftor/ivacaftor combination.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Homozygote; Humans; Mutation | 2023 |
Novel tricyclic pyrrolo-quinolines as pharmacological correctors of the mutant CFTR chloride channel.
Topics: Aminopyridines; Benzodioxoles; Chloride Channels; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation; Quinolines | 2023 |
Computational Exploration of Potential CFTR Binding Sites for Type I Corrector Drugs.
Topics: Aminopyridines; Benzodioxoles; Binding Sites; Cryoelectron Microscopy; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Ligands; Mutation | 2023 |
Real-life experience with a generic formulation of lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for the Phe508del CFTR mutation.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Humans; Male; Mutation | 2023 |