alpha-aminopyridine has been researched along with Cystic Fibrosis of Pancreas in 294 studies
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 1 (0.34) | 29.6817 |
| 2010's | 185 (62.93) | 24.3611 |
| 2020's | 108 (36.73) | 2.80 |
| Authors | Studies |
|---|---|
| Chróinín, MN; Croinin, K; Crowley, J; Mullane, D | 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 |
| Chen, J; Fiedorczuk, K | 1 |
| Zhang, B; Zhang, S | 1 |
| Burgel, PR; Campredon, A; Chassagnon, G; Vakalopoulou, M | 1 |
| Kim, M; McDonald, EF; Plate, L; Sabusap, CMP | 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 |
| Brunsveld, L; Carlile, GW; Chakka, SK; Gielkens, F; Hanrahan, JW; Macdonald, D; Ottmann, C; Peterson, ML; Richard, L; Stevers, LM; Thomas, DY; Thomas, H; Wolter, M | 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 |
| D'Ursi, P; Ford, RC; Fossa, P; Meng, X; Milanesi, M; Orro, A; Padoan, R; Pedemonte, N; Pesce, E; Rondina, A; Rusnati, M; Uggeri, M; Urbinati, C | 1 |
| Amaral, MD; Bacalhau, M; Buarque, CD; da Silva, VD; Ferreira, FC; Kmit, A; Lopes-Pacheco, M; Pimentel, AS; Souza, FR | 1 |
| Yang, C | 1 |
| Perrem, L; Ratjen, F | 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 |
| Chamness, LM; Kuntz, CP; McDonald, EF; McKee, AG; Meiler, J; Noguera, K; Oliver, KE; Penn, WD; Plate, L; Roushar, FJ; Schlebach, JP | 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 |
| Beiersdorf, N; Groten, T; Jaudszus, A; Lorenz, M; Mainz, JG; Michl, RK; Schneider, U | 1 |
| Bell, SC; Fridman, M; Kinnman, N; Macey, J; MacGregor, G; Madge, S; Mainz, JG; Narayanan, S; Suthoff, ED | 1 |
| Connett, GJ | 1 |
| Ahmadi, S; Bartlett, C; Bear, CE; Gonska, T; Ip, W; Jiang, J; Laselva, O; Lew, A; Moraes, TJ; Ouyang, H; Wellhauser, L; Wu, YS; Xia, S | 1 |
| Alper, CJ; Bacon, R; Greenwood, BC; Jeffrey, PL; Lenz, K; Stevens, K; Tesell, MA | 1 |
| Fitch, J; Hayes, D; Jaramillo, L; Kopp, BT; Mejias, A; Palacios, S; Partida-Sanchez, S; Ramilo, O; Robledo-Avila, F; Shrestha, CL; White, P; Woodley, F; Zhang, S | 1 |
| Bellaire, S; Bwirire, D; Conrath, K; Corveleyn, S; Fischer, R; Gleiber, W; Hector, A; Kempa, A; Muller, K; Pano, A; Santermans, E; Schwarz, C; Sutharsan, S; Van de Steen, O; van Koningsbruggen-Rietschel, S; Van Osselaer, N | 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 |
| Hollis, A | 1 |
| Jain, M; Sala, MA | 2 |
| Cuyx, S; De Boeck, K | 1 |
| Chou, FY; Hamilton, CM; Lam, GY; Quon, BS; Sergeev, V; Wilcox, PG | 1 |
| Douros, K; Loukou, I; Moustaki, M; Plyta, M | 1 |
| Amico, G; Baroni, D; Brandas, C; Moran, O | 1 |
| Ehrhardt, A; Hong, JS; Joshi, D; Sorscher, EJ | 1 |
| Barker, D; Burr, L; Dorahy, D; France, M; Greville, H; Middleton, P; Tong, K; Visser, S; Wainwright, C; Wark, P | 1 |
| Mall, MA; Mayer-Hamblett, N; Rowe, SM | 1 |
| Deber, CM; Hartmann, A; Krainer, G; Ravamehr-Lake, D; Schenkel, M; Schlierf, M | 1 |
| Durieu, I; Garcia, S; Nave, V; Olivereau, L; Perceval, M; Rabilloud, M; Reynaud, Q | 1 |
| Carson, S; Christianson, MS; Claudio, AT; Lechtzin, N; Montemayor, K; West, NE | 1 |
| Cryan, SA; Cutrona, MB; De Santi, C; Fernández Fernández, E; Gaul, R; Glasgow, A; Greene, CM; Harvey, BJ; Hawkins, F; Henshall, DC; Hurley, K; Linnane, B; MacLoughlin, R; McNally, P; Mitash, N; Mu, F; Oglesby, IK; Raoof, R; Simpson, JC; Swiatecka-Urban, A; Vencken, S | 1 |
| Bear, CE; Deber, CM; Laselva, O; Stone, TA | 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 |
| Daniels, MLA; Ebert, CS; Farzal, Z; Kimple, AJ; Lee, SE; Senior, BA; Thorp, BD; Zanation, AM | 1 |
| Fajac, I; Girodon, E | 1 |
| Abely, M; Bounyar, L; Chatron, E; Chiron, R; Cosson, L; Dominique, S; Durieu, I; Gerardin, M; Kessler, L; Kessler, R; Mankikian, J; Melly, L; Misgault, B; Porzio, M; Reynaud, Q; Ronsin-Pradel, O; Touzet, S; Troussier, F; Weiss, L | 1 |
| Cottrill, KA; Farinha, CM; McCarty, NA | 1 |
| Doull, I | 2 |
| 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 |
| Aalbers, BL; Arets, HGM; Bronsveld, I; de Kiviet, AC; de Winter-de Groot, KM; Heijerman, HGM; Hofland, RW; Kruijswijk, MA; Michel, S; Schotman, S; van der Ent, CK; van Oirschot-van de Ven, MMM | 1 |
| Al Shadfan, LM; Altıntaş, DU; Aslan, AT; Başaran, AE; Bingöl, A; Çakır, E; Çaltepe, G; Can, D; Çekiç, Ş; Çelebioğlu, E; Cinel, G; Çobanoğlu, N; Çokuğraş, H; Demir, E; Doğru, D; Emiralioğlu, N; Ercan, Ö; Gürsoy, TR; Hangül, M; Harmancı, K; Karagöz, D; Kartal Öztürk, G; Kılıç, M; Kılınç, AA; Kiper, N; Korkmaz Ekren, P; Köse, M; Özcan, G; Özçelik, U; Özdemir, A; Özdoğan, Ş; Pekcan, S; Sapan, N; Şen, HS; Şen, V; Şişmanlar Eyüboğlu, T; Süleyman, A; Tamay, Z; Topal, E; Tuğcu, G; Yalçın, E; Yazan, H; Yıldırım, GK; Yılmaz, Ö; Yüksel, H | 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 |
| Cholon, DM; Esther, CR; Gentzsch, M; Guhr Lee, TN; Quinney, NL | 1 |
| Caputo, M; Carnovale, V; Castaldo, G; Cernera, G; Comegna, M; Corso, G; Gelzo, M; Iacotucci, P | 1 |
| George, A; Goetz, DM; Sawicki, GS; Smith, B | 1 |
| Brown, J; de Vries, JM; Fabbrini, AL; Green, D; Kidder, M; Kucera, JN; Wilsey, M | 1 |
| Abely, M; Biouhee, T; Bui, S; Burgel, PR; Chiron, R; Corvol, H; Dehillotte, C; Durieu, I; Hubert, D; Lemonnier, L; Macey, J; Marguet, C; Martin, C; Mely, L; Munck, A; Murris-Espin, M; Paillasseur, JL; Porzio, M; Prevotat, A; Reix, P; Sermet-Gaudelus, I; Silva, JD | 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 |
| Finnegan, R; O'Grady, E; Ryan, S; Smyth, A; Williamson, M | 1 |
| Aridgides, DS; Armstrong, DA; Ashare, A; Dessaint, JA; Hampton, TH; Hazlett, HF; Mellinger, DL; Nymon, AB | 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 |
| Armirotti, A; Bandiera, T; Bertozzi, SM; Braccia, C; Giraudo, A; Liessi, N; Pedemonte, N; Pesce, E | 1 |
| Finkbeiner, WE; Haggie, PM; Janahi, IA; Nielson, DW; Phuan, PW; Rivera, AA; Tan, JA; Thomas, MM; Verkman, AS | 1 |
| Arest, CF; De Jong, E; Falsafi, R; Garratt, LW; Hancock, REW; Hillas, J; Iosifidis, T; Kicic, A; Kicic-Starcevich, E; Lannigan, FJ; Lee, AHY; Ling, KM; Looi, K; Martinovich, KM; Montgomery, ST; Shaw, NC; Smith, ML; Stick, SM; Sutanto, EN; Vijayasekaran, S | 1 |
| Beekman, JM; Graeber, SY; Hirtz, S; Kruisselbrink, E; Mall, MA; van der Ent, CK; van Mourik, P; Vonk, AM | 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 |
| Bartlett, C; Bear, CE; Gonska, T; Gunawardena, TNA; Laselva, O; Moraes, TJ; Ouyang, H; Popa, A | 1 |
| Xia, X | 1 |
| Ejiofor, LCK; Jensen-Fangel, S; Mathiesen, IHM; Olesen, HV; Pedersen, CL; Philipsen, LKD; Pressler, T; Skov, M | 1 |
| Antos, N; Beisang, D; Billings, J; Frederick, C; Heltshe, SL; Mann, M; Moheet, A; Moran, A; Rowe, SM; Sagel, SD; VanDalfsen, JM; Zhang, L | 1 |
| Edelman, A; Gauthier, S; Guerrera, IC; Hinzpeter, A; Jung, V; Martignetti, L; Nguyen-Khoa, T; Pranke, I; Semeraro, M; Sermet-Gaudelus, I; Stoven, V | 1 |
| Casement, J; Delpiano, L; Gray, MA; Lin, J; Onuora, JC; Saint-Criq, V | 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 |
| Choudhury, P; Colecraft, HM; Jain, A; Kanner, SA; Shuja, Z | 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 |
| Cagnina, RE; Sawicki, GS | 1 |
| Dave, K; Davies, JC; Dobra, R; Matthews, J; Saunders, C; Scott, S; Simmonds, NJ | 1 |
| VanDevanter, DR | 1 |
| Kuntz, KM; Pearson, SD; Rind, DM; Seidner, M; Tice, JA; Wherry, K | 1 |
| Chilvers, MA; Cornell, AG; Davies, JC; Han, Z; Milla, C; Owen, CA; Ratjen, F; Tian, S | 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 |
| Clancy, JP; Konstan, MW; Mayer-Hamblett, N; Odem-Davis, K; Rowe, SM; Skalland, M; VanDevanter, DR; Zemanick, ET | 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 |
| Perkins, RC; Sawicki, GS; Shah, M | 1 |
| Lebecque, P; Thimmesch, M | 1 |
| Amato, F; Castaldo, G; Castellani, C; Ferrari, B; Galietta, LJV; Taccetti, G; Terlizzi, V | 1 |
| Course, CW; Doull, I; Edmondson, C | 1 |
| Franceschelli, S; Pascale, M; Pecoraro, M | 1 |
| Bertini, M; Casiraghi, A; Cipolli, M; Fedrigo, A; Iansa, P; Kleinfelder, K; Melotti, P; Minghetti, P; Pintani, E; Sorio, C; Treggiari, D; Tridello, G | 1 |
| Chilvers, M; Cornell, AG; Hoppe, JE; McColley, SA; McNamara, JJ; Owen, CA; Ratjen, F; Tian, S; Zahigian, R | 1 |
| Baettig, U; Bahra, P; Bala, K; Brocklehurst, C; Budd, E; Butler, R; Cheung, AK; Choudhury, H; Collingwood, SP; Cox, B; Danahay, H; Edwards, L; Everatt, B; Glaenzel, U; Glotin, AL; Gosling, M; Grand, DL; Groot-Kormelink, P; Hall, E; Hatto, J; Howsham, C; Hughes, G; King, A; Koehler, J; Kulkarni, S; Lightfoot, M; Nicholls, I; Page, C; Pergl-Wilson, G; Popa, MO; Robinson, R; Rowlands, D; Sandham, DA; Sharp, T; Spendiff, M; Stanley, E; Steward, O; Taylor, RJ; Tranter, P; Wagner, T; Watson, H; Williams, G; Wright, P; Young, A | 1 |
| Bennett, WD; Bhambhvani, PG; Ceppe, A; Clancy, JP; Corcoran, TE; Donaldson, SH; Laube, BL; Mogayzel, P; Pilewski, JM; Ratjen, F; Rowe, SM; Sagel, SD; Wu, J | 1 |
| Bessaci-Kabouya, K; Bokov, P; Bui, S; Burgel, PR; Choukroun, ML; Da Silva, J; Gerardin, M; Ioan, I; Le Bourgeois, M; Paillasseur, JL; Reix, P; Tatopoulos, A | 1 |
| Hafkemeyer, S; Nietert, MM; Stanke, F; Vinhoven, L | 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 |
| Egan, ME | 1 |
| Braun, A; Dezube, R; Grant, J; Hong, G; Jennings, MT; Lechtzin, N; Merlo, CA; Paranjape, S; West, NE | 1 |
| Barry, P; Horsley, A | 1 |
| Ramsey, BW; Welsh, MJ | 1 |
| Goralski, JL; McKinzie, CJ; Noah, TL; Prieur, MB; Retsch-Bogart, GZ | 1 |
| Mayer-Hamblett, N; VanDevanter, DR | 1 |
| Casciaro, R; Costa, S; Dang, P; Majo, F; Ros, M | 1 |
| Houwen, RHJ; Slae, M; van der Woerd, WL; Wilschanski, M | 1 |
| Amenduni, M; Fabris, L; Fiorotto, R; Mariotti, V; Spirli, C; Strazzabosco, M | 1 |
| Bertolini, A; Bodewes, FAJA; Jonker, JW; van de Peppel, IP; Verkade, HJ | 1 |
| McColley, SA; Talamo Guevara, M | 1 |
| Davies, J; Elborn, JS | 1 |
| Bulloch, MN; Giovane, R; Hanna, C | 1 |
| AbuArish, A; Cantin, AM; Cloutier, A; Cosa, G; Greene, LE; Hanrahan, JW; Matthes, E; Robert, R; Thomas, DY; Turner, MJ; Wong, FH | 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 | 2 |
| 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 |
| Cutting, GR | 1 |
| Amer, AO; Badr, A; Caution, K; Cormet-Boyaka, E; Dakhlallah, D; Hall-Stoodley, L; Hamilton, K; Hayes, D; Kirkby, SE; Klamer, BG; Kopp, BT; Krause, K; McCoy, KS; Parinandi, NL; Partida-Sanchez, S; Robledo-Avila, F; Shrestha, C; Tazi, MF; Tumin, D; Zhang, X | 1 |
| Campbell, JD; Dilokthornsakul, P; Patidar, M | 1 |
| DiMango, E; Hopkins, BD; Kitur, K; Parsons, R; Prince, A; Riquelme, SA; Wolfe, AL | 1 |
| Alejandre Alcazar, MA; Mueller, MI; Rietschel, E; Thomassen, JC; van Koningsbruggen-Rietschel, S | 1 |
| Baatallah, N; Bitam, S; Callebaut, I; Chevalier, B; Costes, B; Edelman, A; Fanen, P; Girodon, E; Hinzpeter, A; Hoffmann, B; Martin, N; Mekki, C; Mornon, JP; Pranke, I; Sermet-Gaudelus, I; Servel, N; Simonin, J | 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 |
| Bagheri-Hanson, A; Dopfer, C; Dorda, M; Fischer, S; Graeber, SY; Gyulumyan, L; Hirtz, S; Hyde, R; Mairbäurl, H; Mall, MA; Naehrlich, L; Rueckes-Nilges, C; Scheuermann, H; Tümmler, B; Wege, S | 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 |
| Benden, C; Bürgi, U; Hirt, A; Huber, LC; Kurowski, T; Murer, C; Robinson, CA | 1 |
| Hayes, D; Kopp, BT; McCulloch, S; Sarzynski, L; Shrestha, CL; Woodley, FW; Zhang, S | 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 |
| Schneider, EK | 1 |
| Bear, CE; Casavola, V; Laselva, O; Molinski, S | 2 |
| Adam, D; Bilodeau, C; Brochiero, E; Maillé, É; Ruffin, M; Sognigbé, L | 1 |
| Avolio, J; Bartlett, C; Bear, CE; Du, K; Eckford, PDW; Gonska, T; He, G; Ho, K; Huan, LJ; Jiang, JX; Klingel, M; Kyriakopoulou, L; McCormack, J; Moraes, TJ; Munsie, L; Ouyang, H; Pereira, SL; Ratjen, F; Rossant, J; Stanojevic, S; Strug, LJ; Wellhauser, L; Wong, AP; Yang, JY | 1 |
| Bruch, BA; Ramsey, LJ; Singh, SB; Starner, TD | 1 |
| Gentzsch, M; Mall, MA | 1 |
| Flume, PA; Jennings, MT | 1 |
| Esther, CR; McKinzie, C; Stringer, E; Terrell, M; Trimble, A | 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 |
| Connett, GJ; Hammond, JA | 1 |
| Bergbower, EAS; Boinot, C; Cebotaru, L; Guggino, WB; Liu, Q; Sabirzhanova, I; Yanda, MK | 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 |
| Nevitt, SJ; Patel, S; Sinha, IP; Southern, KW | 2 |
| Chan, CL; Li, A; Nadeau, K; Pyle, L; Sagel, SD; Vigers, T; Zemanick, E | 1 |
| Crawford, KJ; Downey, DG | 1 |
| Boyle, MP; Jiang, JG; Konstan, MW; Marigowda, G; McColley, SA; Ramsey, BW; Rubin, JL; Stuart Elborn, J; Vera-Llonch, M; Wainwright, CE; Waltz, D | 1 |
| Barry, PJ; Jones, AM; Mitchell, RM | 1 |
| Amaral, MD; Barros, P; Faria, M; Gomes-Duarte, A; Jordan, P; Matos, AM; Matos, P | 1 |
| Fink, AK; Loeffler, DR; Mayer-Hamblett, N; Sawicki, GS; Schechter, MS | 1 |
| Kissner, D; Le Camus, C; LeFlore, Y; Marigowda, G; Narayan, SB; Simard, C | 1 |
| Covvey, JR; Giannetti, V; Kamal, KM; Mukherjee, K; Vadagam, 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 |
| Hwang, TC; Yeh, JT; Yu, YC | 1 |
| Kopp, BT; Shrestha, CL; Zhang, S | 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 |
| Bratcher, PE; Nichols, DP; Nick, JA; Pohl, K; Saavedra, MT; Strand, MJ; Taylor-Cousar, JL | 1 |
| Cheng, DY; Wei, J; Wu, HX; Xiong, XF; Zhu, M; Zhuo, KQ | 1 |
| Flume, PA; Kosinski, M; Marigowda, G; Quittner, AL; Suthoff, ED | 1 |
| Achimastos, D; Baatallah, N; Bailly, C; Chedevergne, F; Edelman, A; Flament, T; Girodon, E; Hatton, A; Hinzpeter, A; Kyrilli, S; Le Bourgeois, M; Masson, A; Nguyen-Khoa, T; Schneider-Futschik, EK; Sermet-Gaudelus, I | 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 |
| De Wachter, E; Vanderhelst, E; Verbanck, S; Vincken, S | 1 |
| Middleton, PG | 1 |
| De la Hoz, D; Restrepo-Gualteros, SM; Villamil Osorio, M | 1 |
| Palange, P; Righelli, D; Savi, D; Schiavetto, S; Simmonds, NJ | 1 |
| Alexiou, S; Cheng, PC; Rubenstein, RC | 1 |
| Bratcher, PE; Chioccioli, M; Cicuta, P; Feriani, L; Kotar, J | 1 |
| De Biase, RV; Gnessi, L; Lenzi, A; Lubrano, C; Pascucci, C; Quattrucci, S; Savi, D | 1 |
| Fischer, AJ; Fox, C; Gates, LK; McLearn-Montz, AJ; Milavetz, F; Murry, LT; Porterfield, HS; Sabus, A; Singh, SB | 1 |
| Balfour-Lynn, IM | 1 |
| Bolan, C; Cortese, C; Harnois, D; Mallea, 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 |
| Brannan, J; Cookson, K; Dorahy, DJ; Thiruchelvam, T; Wark, PAB | 1 |
| Burki, TK | 1 |
| Bertoncini, E; Colomb-Lippa, D | 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 |
| 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 | 1 |
| 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 |
| Bompadre, SG; Kopeikin, Z; Yang, HY; Yuksek, Z | 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 |
| 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 |
| Davis, PB | 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 |
| Barry, PJ; Jones, AM | 1 |
| Marshall, SG; Ramsey, BW; Rowe, SM; Solomon, GM | 1 |
| Bilton, D | 1 |
| Amaral, MD; Awatade, NT; Clarke, LA; Farinha, CM; Mendes, K; Pastor, J; Ramos, MM; Solé, A; Uliyakina, I | 1 |
| Becq, F | 1 |
| Nau, JY | 1 |
| Ferkol, T; Quinton, P | 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 |
| Ong, T; Ramsey, BW | 2 |
| Silverman, E | 1 |
| Brodsky, JL; Frizzell, RA | 1 |
| 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 |
| Boyle, M; Elborn, JS; Ramsey, B; Wainwright, C | 1 |
| Elborn, JS; Ramsey, BW; Wainwright, CE | 1 |
| Baloch, NU; Janahi, IA; Rehman, A | 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 |
| Mayer, M | 1 |
| Bui, S; Colomb-Jung, V; Corvol, H; Hubert, D; Marguet, C; Munck, A; Murris-Espin, M | 1 |
| Jeon, DK; Jo, S; Khloya, P; Kumar, S; Lee, HK; Namkung, W; Park, J; Seo, Y; Sharma, PK | 1 |
| Boyle, M; Mayer-Hamblett, N; VanDevanter, D | 1 |
| Reinhardt, D | 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 |
| Meißner, T | 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 |
| 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 |
| Cogen, J; Goss, CH; Heltshe, SL; Ramos, KJ | 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 |
| Goss, CH; Huang, X; Konstan, MW; Lubarsky, B; Marigowda, G; Mayer-Hamblett, N; McKone, EF; Millar, SJ; Morgan, W; Moss, RB; Pasta, DJ; Rubin, J; Sawicki, GS; Tian, S; Waltz, D | 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 |
| Casey, A; Chmiel, JF; Donaldson, SH; Flume, PA; Mandagere, A; McCoy, K; Shoemaker, SA; Solomon, GM; Taylor-Cousar, JL; Troha, JM; Zeitlin, PL; Zemanick, ET | 1 |
| 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 |
| Aleksandrov, AA; Cui, L; Dokholyan, NV; He, L; Jensen, T; Kota, P; Riordan, JR | 1 |
55 review(s) available for alpha-aminopyridine and Cystic Fibrosis of Pancreas
| Article | Year |
|---|---|
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 |
Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Design; Drug Therapy, Combination; Humans; Quinolones | 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 |
The Extrapulmonary Effects of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Mutation; Quinolones; Signal Transduction | 2020 |
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 |
Cystic fibrosis 2019: Year in review.
Topics: Administration, Inhalation; Aminophenols; Aminopyridines; Anti-Bacterial Agents; Azithromycin; Benzodioxoles; Carrier State; Chloride Channel Agonists; Cystic Fibrosis; Drug Combinations; Exercise; Health Services Accessibility; Healthcare Disparities; Humans; Indoles; Inflammation; Physical Therapy Modalities; Pseudomonas Infections; Pyrazoles; Pyridines; Quinolines; Quinolones; Saline Solution, Hypertonic | 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 |
Innovating cystic fibrosis clinical trial designs in an era of successful standard of care therapies.
Topics: Aminophenols; Aminopyridines; Anti-Bacterial Agents; Benzodioxoles; Chloride Channel Agonists; Clinical Trials as Topic; Comparative Effectiveness Research; Cystic Fibrosis; Disease Progression; Drug Combinations; Humans; Pseudomonas Infections; Quinolones; Standard of Care; Treatment Outcome | 2017 |
Effects of new and emerging therapies on gastrointestinal outcomes in cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Body Mass Index; Chloride Channel Agonists; Cystic Fibrosis; Digestive System; Drug Combinations; Gastroesophageal Reflux; Humans; Hydrogen-Ion Concentration; Intestinal Diseases; Liver Diseases; Probiotics; Quinolones; Treatment Outcome; Weight Gain | 2017 |
Diagnosis, follow-up and treatment of cystic fibrosis-related liver disease.
Topics: Aftercare; Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cholagogues and Choleretics; Cystic Fibrosis; Drug Combinations; Elasticity Imaging Techniques; Humans; Liver Diseases; Liver Transplantation; Quinolones; Ursodeoxycholic Acid | 2017 |
The safety of lumacaftor and ivacaftor for the treatment of cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Child; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Cytochrome P-450 CYP3A; Drug Combinations; Drug Interactions; Humans; Mutation; Quinolones | 2017 |
Lumacaftor/ivacaftor, a novel agent for the treatment of cystic fibrosis patients who are homozygous for the F580del CFTR mutation.
Topics: Aminophenols; Aminopyridines; Animals; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Forced Expiratory Volume; Humans; Molecular Targeted Therapy; Mutation; Quinolones | 2017 |
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 |
Cystic fibrosis papers of the year 2017.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Management; Drug Combinations; Humans; Lung; Membrane Transport Modulators; Mutation; Quinolones; Respiratory Function Tests; Time | 2018 |
The CF Canada-Sick Kids Program in individual CF therapy: A resource for the advancement of personalized medicine in CF.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Canada; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genetic Therapy; Humans; Incidence; Mutation, Missense; Precision Medicine; Program Development; Quinolones; RNA | 2019 |
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 |
Cystic Fibrosis: Translating Molecular Mechanisms into Effective Therapies.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Development; Humans; Indoles; Molecular Targeted Therapy; Mutation; 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 |
Tezacaftor for the treatment of cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genotype; Humans; Indoles; Mutation; Quinolones | 2018 |
Theratyping in cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Gene Editing; Genetic Therapy; Genotype; Humans; Indoles; Mutation; Organoids; Oxadiazoles; Precision Medicine; Quinolones | 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 |
Efficacy and Safety of CFTR Corrector and Potentiator Combination Therapy in Patients with Cystic Fibrosis for the F508del-CFTR Homozygous Mutation: A Systematic Review and Meta-analysis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Combined Modality Therapy; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Dose-Response Relationship, Drug; Homozygote; Humans; Mutation; Quinolones | 2019 |
Cystic fibrosis transmembrane conductance regulator modulators: Present and future in cystic fibrosis treatment. A review.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Indoles; Precision Medicine; Quality of Life; Quinolones | 2019 |
Safety and efficacy of treatment with lumacaftor in combination with ivacaftor in younger patients with cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Child, Preschool; Clinical Trials as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Mutation; Quinolones; Treatment Outcome | 2019 |
Clinical papers of the year 2018 - Cystic fibrosis.
Topics: Administration, Inhalation; Aminophenols; Aminopyridines; Anti-Bacterial Agents; Azithromycin; Benzodioxoles; Bronchoalveolar Lavage; Bronchoalveolar Lavage Fluid; Chloride Channel Agonists; Cough; Cross Infection; Cystic Fibrosis; Disease Progression; Drug Combinations; Drug Therapy, Combination; Humans; Indoles; Lung Transplantation; Microbiological Techniques; Mycobacterium abscessus; Mycobacterium Infections, Nontuberculous; Nebulizers and Vaporizers; Proton Pump Inhibitors; Pseudomonas aeruginosa; Pseudomonas Infections; Pyrazoles; Pyridines; Pyrrolidines; Quinolones; Randomized Controlled Trials as Topic; Saline Solution, Hypertonic; Specimen Handling; Sputum; Treatment Outcome; Tuberculosis, Pulmonary | 2020 |
Cystic Fibrosis-Associated Liver Disease in Lung Transplant Recipients.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Incidence; Liver Cirrhosis; Liver Transplantation; Lung Transplantation; Mutation; Postoperative Care; Quinolones; Survival Rate; Transplant Recipients; Treatment Outcome; Ursodeoxycholic Acid | 2019 |
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 |
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 |
[New therapies for cystic fibrosis targeting the CFTR gene or the CFTR protein].
Topics: Age Factors; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genetic Therapy; Humans; Molecular Targeted Therapy; Quinolones | 2016 |
[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 |
Advancing clinical development pathways for new CFTR modulators in cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Clinical Trials as Topic; Critical Pathways; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genetic Therapy; Genotype; Humans; Molecular Targeted Therapy; Mutation; Quality of Life; Quinolones; Randomized Controlled Trials as Topic; 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 |
Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Approval; Drug Combinations; Humans; Quinolones; United States; United States Food and Drug Administration | 2017 |
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 |
13 trial(s) available for alpha-aminopyridine and Cystic Fibrosis of Pancreas
| Article | Year |
|---|---|
GLPG2737 in lumacaftor/ivacaftor-treated CF subjects homozygous for the F508del mutation: A randomized phase 2A trial (PELICAN).
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Homozygote; Humans; Male; Mutation; Quinolones; Respiratory Function Tests; Sweat; Treatment Outcome | 2020 |
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 |
Patients with cystic fibrosis and advanced lung disease benefit from lumacaftor/ivacaftor treatment.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Forced Expiratory Volume; Humans; Lung; Male; Middle Aged; Quality of Life; Quinolones; 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 |
Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study.
Topics: Aminophenols; Aminopyridines; Australia; Benzodioxoles; Canada; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Europe; Female; Humans; Male; Mutation; Quinolones; Time; Treatment Outcome; United States | 2021 |
Lumacaftor/Ivacaftor reduces pulmonary exacerbations in patients irrespective of initial changes in FEV
Topics: Adolescent; Adult; Aged; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Disease Progression; Double-Blind Method; Drug Combinations; Female; Follow-Up Studies; Forced Expiratory Volume; Humans; Lung; Male; Middle Aged; Quinolones; Respiratory Function Tests; Retrospective Studies; Treatment Outcome; Young Adult | 2019 |
Measuring recovery in health-related quality of life during and after pulmonary exacerbations in patients with cystic fibrosis.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Disease Progression; Drug Combinations; Female; Humans; Male; Quality of Life; Quinolones; Recovery of Function; Time Factors; Treatment Outcome | 2019 |
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 |
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 |
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cough; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Progression; Double-Blind Method; Drug Administration Schedule; Drug Combinations; Female; Forced Expiratory Volume; Homozygote; Humans; Lung; Male; Mutation; Quinolones; Time; Treatment Outcome; Young Adult | 2017 |
Pharmacokinetics and safety of cavosonstat (N91115) in healthy and cystic fibrosis adults homozygous for F508DEL-CFTR.
Topics: Adult; Aldehyde Oxidoreductases; Aminophenols; Aminopyridines; Benzodioxoles; Biological Availability; Biphenyl Compounds; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Cytochrome P-450 CYP3A Inducers; Dose-Response Relationship, Drug; Drug Combinations; Drug Interactions; Drug Monitoring; Female; Humans; Male; Membrane Transport Modulators; Mutation; Pharmacogenetics; Quinolones; Rifampin; Treatment Outcome | 2017 |
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 |
226 other study(ies) available for alpha-aminopyridine and Cystic Fibrosis of Pancreas
| Article | Year |
|---|---|
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 |
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 |
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 |
Distinct proteostasis states drive pharmacologic chaperone susceptibility for cystic fibrosis transmembrane conductance regulator misfolding mutants.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation; Proteostasis; Signal Transduction | 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 |
Macrocycle-stabilization of its interaction with 14-3-3 increases plasma membrane localization and activity of CFTR.
Topics: Aminophenols; Aminopyridines; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation | 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 |
Virtual Drug Repositioning as a Tool to Identify Natural Small Molecules That Synergize with Lumacaftor in F508del-CFTR Binding and Rescuing.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Repositioning; Humans; Mutation; Niacinamide; Proteasome Endopeptidase Complex; Ubiquitins | 2022 |
Identification of novel F508del-CFTR traffic correctors among triazole derivatives.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation; Triazoles | 2023 |
SIMPLIFYing cystic fibrosis treatment in a post-modulator era.
Topics: Aminophenols; Aminopyridines; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation | 2023 |
A new triple combination cystic fibrosis transmembrane regulator modulator.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation | 2023 |
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 |
General trends in the effects of VX-661 and VX-445 on the plasma membrane expression of clinical CFTR variants.
Topics: Aminopyridines; Cell Membrane; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Mutation | 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 |
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 |
Patient-reported outcomes in patients with cystic fibrosis with a G551D mutation on ivacaftor treatment: results from a cross-sectional study.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Cross-Sectional Studies; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Forced Expiratory Volume; Humans; Internationality; Male; Multivariate Analysis; Mutation; Patient Reported Outcome Measures; Quality of Life; Quinolones; Regression Analysis; Surveys and Questionnaires | 2019 |
ORKAMBI-Mediated Rescue of Mucociliary Clearance in Cystic Fibrosis Primary Respiratory Cultures Is Enhanced by Arginine Uptake, Arginase Inhibition, and Promotion of Nitric Oxide Signaling to the Cystic Fibrosis Transmembrane Conductance Regulator Channe
Topics: Aminophenols; Aminopyridines; Animals; Arginase; Arginine; Benzodioxoles; Bronchi; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Cytosol; Drug Combinations; Humans; Intestinal Mucosa; Mice; Mutation; Nitric Oxide; Nose; 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 |
Whole-blood transcriptomic responses to lumacaftor/ivacaftor therapy in cystic fibrosis.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Biomarkers, Pharmacological; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Homozygote; Humans; Ion Transport; Male; Metabolomics; Mutation; Pharmacogenomic Testing; Pharmacogenomic Variants; Prognosis; Quinolones; Transcriptome | 2020 |
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 |
Orphan Drug Pricing and Costs: A Case Study of Kalydeco and Orkambi.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cost-Benefit Analysis; Cystic Fibrosis; Drug Combinations; Drug Costs; Humans; Orphan Drug Production; Quinolones | 2019 |
Combination Therapy with Lumacaftor-Ivacaftor in Cystic Fibrosis. Keeping It Real.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Humans; Quinolones | 2020 |
Longitudinal changes in lung function following initiation of lumacaftor/ivacaftor combination.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Monitoring; Duration of Therapy; Female; Forced Expiratory Volume; Humans; Longitudinal Studies; Male; Medication Therapy Management; Quinolones; Respiratory Function Tests; Retrospective Studies; Treatment Outcome; United States | 2020 |
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 |
Lumacaftor/ivacaftor reduces exacerbations in adults homozygous for Phe508del mutation with severe lung disease.
Topics: Administration, Intravenous; Adult; Aminophenols; Aminopyridines; Anti-Bacterial Agents; Australia; Benzodioxoles; Case-Control Studies; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug-Related Side Effects and Adverse Reactions; Female; Forced Expiratory Volume; Humans; Male; Mutation; Quinolones; Respiratory Function Tests; Respiratory Tract Infections; Symptom Flare Up | 2020 |
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 |
Adherence to lumacaftor-ivacaftor therapy in patients with cystic fibrosis in France.
Topics: Adult; Age Factors; Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cost-Benefit Analysis; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Forced Expiratory Volume; France; Homozygote; Humans; Male; Medication Adherence; Quinolones; Respiratory Function Tests; Retrospective Studies; Treatment Outcome | 2020 |
Unmasking catamenial hemoptysis in the era of CFTR modulator therapy.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Bronchoscopy; Chloride Channel Agonists; Cystic Fibrosis; Drug Combinations; Drug Therapy, Combination; Endometriosis; Female; Fertility Agents, Female; Hemoptysis; Humans; Indoles; Leuprolide; Pyrazoles; Pyridines; Quinolines; Quinolones; Radiography, Thoracic; Thoracic Diseases; Tomography, X-Ray Computed | 2020 |
Precise Targeting of miRNA Sites Restores CFTR Activity in CF Bronchial Epithelial Cells.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Bronchi; Cells, Cultured; Child; Child, Preschool; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Synergism; Epithelial Cells; Female; Humans; Indoles; Infant; Male; MicroRNAs; Middle Aged; Models, Biological; Nanoparticles; Oligonucleotides; Polylactic Acid-Polyglycolic Acid Copolymer; Quinolones | 2020 |
Anti-Infectives Restore ORKAMBI
Topics: Aminophenols; Aminopyridines; Anti-Bacterial Agents; Antimicrobial Cationic Peptides; Benzodioxoles; Bronchi; Cell Line; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Mutation; Pseudomonas aeruginosa; Pseudomonas Infections; Quinolones; Tobramycin | 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 |
Genomically-guided therapies: A new era for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genetic Therapy; Humans; Indoles; Mutation; Precision Medicine; Quinolones; Respiratory System Agents | 2020 |
Effect of one-year lumacaftor-ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Blood Glucose; Child; Cystic Fibrosis; Diabetes Mellitus; Drug Administration Schedule; Drug Combinations; Female; Glucose Intolerance; Glucose Tolerance Test; Humans; Male; Middle Aged; Prospective Studies; Quinolones; Young Adult | 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 |
Females with cystic fibrosis have a larger decrease in sweat chloride in response to lumacaftor/ivacaftor compared to males.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Body Mass Index; Child; Chlorides; Correlation of Data; Cystic Fibrosis; Drug Combinations; Female; Forced Expiratory Volume; Humans; Male; Quinolones; Retrospective Studies; Sex Factors; Sweat; Young Adult | 2021 |
Patients eligible for modulator drugs: Data from cystic fibrosis registry of Turkey.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Child, Preschool; Chloride Channel Agonists; Cross-Sectional Studies; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Humans; Indoles; Infant; Male; Mutation; Pyrazoles; Pyridines; Quinolines; Quinolones; Registries; Turkey; Young Adult | 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 |
Accumulation and persistence of ivacaftor in airway epithelia with prolonged treatment.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Bronchi; Cell Culture Techniques; Chloride Channel Agonists; Cystic Fibrosis; Drug Combinations; Epithelial Cells; Humans; Indoles; Quinolones; Respiratory Mucosa | 2020 |
Lumacaftor/ivacaftor improves liver cholesterol metabolism but does not influence hypocholesterolemia in patients with cystic fibrosis.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cholesterol; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Humans; Liver; Male; Mutation; Quinolones; Treatment Outcome; Young Adult | 2021 |
Survey of patients with cystic fibrosis and caregivers decisions regarding CFTR modulators.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Caregivers; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Substitution; Foundations; Humans; Indoles; Middle Aged; Quinolones; Social Media; Surveys and Questionnaires; Young Adult | 2020 |
Cystic Fibrosis-Related Pancreatic Cysts Decrease in Size and Number Upon Treatment With Cystic Fibrosis Transmembrane Conductance Regulator Modulators.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Indoles; Male; Mutation; Pancreas; Pancreatic Cyst; Quinolones; Treatment Outcome; Ultrasonography | 2020 |
Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Disease Progression; Drug Combinations; Female; France; Humans; Male; Quinolones; Respiratory Function Tests | 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 |
Evidence of Small Airways Disease and the Immediate Effects of Lumacaftor/Ivacaftor in Children with Cystic Fibrosis.
Topics: Adolescent; Airway Remodeling; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Drug Combinations; Female; Forced Expiratory Volume; Humans; Male; Negative Results; Pulmonary Disease, Chronic Obstructive; Quinolones; Retrospective Studies; Risk Factors | 2020 |
Altered iron metabolism in cystic fibrosis macrophages: the impact of CFTR modulators and implications for Pseudomonas aeruginosa survival.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Biofilms; Child; Culture Media, Conditioned; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Gene Expression Profiling; Host-Pathogen Interactions; Humans; Iron; Iron-Regulatory Proteins; Macrophages; Male; Middle Aged; Pseudomonas aeruginosa; Quinolones; Sputum | 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 |
Distinctive lipid signatures of bronchial epithelial cells associated with cystic fibrosis drugs, including Trikafta.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Bronchi; Cells, Cultured; Ceramides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Epithelial Cells; Humans; Indoles; Lipid Metabolism; Lipidomics; Pyrazoles; Pyridines; Quinolines; Quinolones; Spectrometry, Mass, Electrospray Ionization | 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 |
Ivacaftor or lumacaftor/ivacaftor treatment does not alter the core CF airway epithelial gene response to rhinovirus.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cells, Cultured; Common Cold; Cystic Fibrosis; Drug Combinations; Humans; Quinolones; Respiratory Mucosa; Rhinovirus | 2021 |
Comparison of Organoid Swelling and
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chlorides; Colforsin; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Edema; Female; Forced Expiratory Volume; Homozygote; Humans; In Vitro Techniques; Male; Membrane Potentials; Middle Aged; Nasal Mucosa; Organoids; Precision Medicine; Prospective Studies; Quinolones; Rectum; Sweat; Treatment Outcome; Vital Capacity; Young Adult | 2020 |
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 |
Emerging preclinical modulators developed for F508del-CFTR have the potential to be effective for ORKAMBI resistant processing mutants.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cells, Cultured; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Evaluation, Preclinical; Drug Resistance; Humans; Mutation; Quinolones | 2021 |
Drug efficacy and toxicity prediction: an innovative application of transcriptomic data.
Topics: Aminophenols; Aminopyridines; Animals; Antineoplastic Agents; Benzodioxoles; Cell Death; Cystic Fibrosis; Dose-Response Relationship, Drug; Drug Combinations; Drug Development; Epithelial Cells; Gene Expression Profiling; Gene Regulatory Networks; Humans; Leukemia, Myeloid, Acute; Pyrazines; Pyrazoles; Quinolones; Toxicity Tests; Transcriptome | 2020 |
Lumacaftor/ivacaftor therapy fails to increase insulin secretion in F508del/F508del CF patients.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Glucose Tolerance Test; Homozygote; Humans; Insulin Secretion; Longitudinal Studies; Male; Middle Aged; Mutation; Quinolones | 2021 |
Urinary Exosomes of Patients with Cystic Fibrosis Unravel CFTR-Related Renal Disease.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Case-Control Studies; Child; Child, Preschool; Cystic Fibrosis; Drug Combinations; Exosomes; Humans; Indoles; Kidney Diseases; Proteome; Quinolones; Young Adult | 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 |
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 |
Targeted deubiquitination rescues distinct trafficking-deficient ion channelopathies.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Channelopathies; Cystic Fibrosis; Deubiquitinating Enzymes; Drug Combinations; Humans; Indoles; Ion Transport; Long QT Syndrome; Myocytes, Cardiac; Nedd4 Ubiquitin Protein Ligases; Potassium Channels; Pyrazoles; Pyridines; Quinolines; Quinolones | 2020 |
The PROSPECT Is Bright for CFTR Modulators.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Quinolones; Treatment Outcome | 2021 |
Tribulations and (clinical) trials in cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cannabinoid Receptor Agonists; Chloride Channel Agonists; Clinical Trials as Topic; Cystic Fibrosis; Dronabinol; Drug Combinations; Humans; Indoles; Quinolones | 2021 |
The march towards CFTR modulator access for all people with CF: The end of the beginning.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Indoles; Mutation; Precision Medicine; Pyrazoles; Pyridines; Pyrrolidines; Quinolones | 2021 |
The effectiveness and value of novel treatments for cystic fibrosis.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cost-Benefit Analysis; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Approval; Drug Combinations; Drug Costs; Health Policy; Humans; Indoles; Models, Economic; Mutation; Pyrazoles; Pyridines; Quinolines; Quinolones; Treatment Outcome; United States; United States Food and Drug Administration | 2021 |
Measuring the impact of CFTR modulation on sweat chloride in cystic fibrosis: Rationale and design of the CHEC-SC study.
Topics: Adolescent; Adult; Aged; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Chlorides; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Humans; Male; Middle Aged; Prospective Studies; Quinolones; Sweat | 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 |
An evaluation of healthcare utilization and clinical charges in children and adults with cystic fibrosis.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cohort Studies; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Patient Acceptance of Health Care | 2021 |
[Cystic fibrosis prognosis in Europa : chronicle of an announced dilemma].
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Europe; Humans; Mutation; Prognosis; Quinolones | 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 |
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 |
Optical Measurements of Sweat for in Vivo Quantification of CFTR Function in Individual Sweat Glands.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Optics and Photonics; Quinolones; Sweat; Sweat Glands | 2021 |
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 |
Discovery of Icenticaftor (QBW251), a Cystic Fibrosis Transmembrane Conductance Regulator Potentiator with Clinical Efficacy in Cystic Fibrosis and Chronic Obstructive Pulmonary Disease.
Topics: Administration, Oral; Aminopyridines; Animals; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Models, Animal; Drug Evaluation, Preclinical; Gene Deletion; Half-Life; Humans; Protein Binding; Pulmonary Disease, Chronic Obstructive; Rats; Rats, Sprague-Dawley; Solubility; Structure-Activity Relationship | 2021 |
Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cohort Studies; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Humans; Longitudinal Studies; Male; Middle Aged; Mucociliary Clearance; Prospective Studies; Quinolones; Young Adult | 2022 |
Real-world assessment of LCI following lumacaftor-ivacaftor initiation in adolescents and adults with cystic fibrosis.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cohort Studies; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Quinolones; Respiratory Function Tests | 2022 |
CFTR Lifecycle Map-A Systems Medicine Model of CFTR Maturation to Predict Possible Active Compound Combinations.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Mutation; Protein Interaction Maps; Systems Analysis | 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 |
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 |
An Observational Study of Outcomes and Tolerances in Patients with Cystic Fibrosis Initiated on Lumacaftor/Ivacaftor.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Dyspnea; Female; Forced Expiratory Volume; Humans; Logistic Models; Lung; Male; Middle Aged; Mutation; Quinolones; Retrospective Studies; Young Adult | 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 |
AJRCCM: 100-Year Anniversary. Progress along the Pathway of Discovery Leading to Treatment and Cure of Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Anniversaries and Special Events; Anti-Bacterial Agents; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Enzyme Replacement Therapy; History, 20th Century; History, 21st Century; Mutation; Periodicals as Topic; Pulmonary Medicine; Quinolones | 2017 |
Worsening anxiety and depression after initiation of lumacaftor/ivacaftor combination therapy in adolescent females with cystic fibrosis.
Topics: Adolescent; Aminophenols; Aminopyridines; Antidepressive Agents; Anxiety; Benzodioxoles; Child; Cognitive Behavioral Therapy; Cystic Fibrosis; Depression; Drug Combinations; Female; Humans; Membrane Transport Modulators; Quinolones; Suicidal Ideation; Suicide, Attempted; Withholding Treatment | 2017 |
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 |
Clinical trial research in focus: ensuring new cystic fibrosis drugs fulfil their potential.
Topics: Aminophenols; Aminopyridines; Anti-Bacterial Agents; Benzodioxoles; Chloride Channel Agonists; Clinical Trials as Topic; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Expectorants; Genetic Therapy; Humans; Indoles; Mutation; Patient Selection; Quality of Life; Quinolones | 2017 |
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 |
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 |
Treating Specific Variants Causing Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genetic Variation; Humans; Quinolones | 2017 |
The expression of Mirc1/Mir17-92 cluster in sputum samples correlates with pulmonary exacerbations in cystic fibrosis patients.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Chloride Channel Agonists; Correlation of Data; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Progression; Drug Combinations; Drug Monitoring; Female; Gene Expression Profiling; Humans; Male; MicroRNAs; Nerve Tissue Proteins; Quinolones; Respiratory Function Tests; Respiratory System; RNA, Long Noncoding; Sputum | 2018 |
Forecasting the Long-Term Clinical and Economic Outcomes of Lumacaftor/Ivacaftor in Cystic Fibrosis Patients with Homozygous phe508del Mutation.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Forced Expiratory Volume; Homozygote; Humans; Markov Chains; Mutation; Quality-Adjusted Life Years; Quinolones; Severity of Illness Index; Treatment Outcome; United States | 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 |
Effect of Lumacaftor/Ivacaftor on glucose metabolism and insulin secretion in Phe508del homozygous cystic fibrosis patients.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Blood Glucose; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Glucose Tolerance Test; Homozygote; Humans; Insulin Secretion; Male; Quinolones; Young Adult | 2018 |
Cis variants identified in F508del complex alleles modulate CFTR channel rescue by small molecules.
Topics: Alleles; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Humans; Mutation; Phenylalanine; Quinolones | 2018 |
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 |
Effects of Lumacaftor-Ivacaftor Therapy on Cystic Fibrosis Transmembrane Conductance Regulator Function in Phe508del Homozygous Patients with Cystic Fibrosis.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Child; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Germany; Homozygote; Humans; Male; Prospective Studies; Quinolones; Young Adult | 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 |
First experience in Switzerland in Phe508del homozygous cystic fibrosis patients with end-stage pulmonary disease enrolled in a lumacaftor-ivacaftor therapy trial - preliminary results.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Genotype; Homozygote; Humans; Lung Transplantation; Male; Mutation; Prospective Studies; Quinolones; Severity of Illness Index; Switzerland | 2018 |
Metabolomic responses to lumacaftor/ivacaftor in cystic fibrosis.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers; Chromatography, Liquid; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Humans; Male; Metabolomics; Middle Aged; Quinolones; Treatment Outcome; Young Adult | 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 |
Cytochrome P450 3A4 Induction: Lumacaftor versus Ivacaftor Potentially Resulting in Significantly Reduced Plasma Concentration of Ivacaftor.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Cytochrome P-450 CYP1A2; Cytochrome P-450 CYP2C9; Cytochrome P-450 CYP3A; Drug Combinations; Drug Interactions; Enzyme Assays; Enzyme Induction; Female; Humans; Indoles; Quinolones | 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 |
CFTR rescue with VX-809 and VX-770 favors the repair of primary airway epithelial cell cultures from patients with class II mutations in the presence of Pseudomonas aeruginosa exoproducts.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cells, Cultured; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Exotoxins; Glycine; Humans; Hydrazines; Mutation; Pseudomonas aeruginosa; Quinolones; Regeneration; Respiratory Mucosa | 2018 |
Impact of a cystic fibrosis transmembrane conductance regulator (CFTR) modulator on high-dose ibuprofen therapy in pediatric cystic fibrosis patients.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Interactions; Drug Therapy, Combination; Female; Humans; Ibuprofen; Male; Quinolones | 2018 |
Measured fetal and neonatal exposure to Lumacaftor and Ivacaftor during pregnancy and while breastfeeding.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Breast Feeding; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Monitoring; Female; Fetal Blood; Humans; Infant, Newborn; Liver Function Tests; Milk, Human; Pregnancy; Pregnancy Complications; Prenatal Exposure Delayed Effects; Quinolones; Treatment Outcome | 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 |
The use of lumacaftor/ivacaftor to treat acute deterioration in paediatric cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Progression; Drug Combinations; Female; Homozygote; Humans; Lung; Membrane Transport Modulators; Mutation; Patient Care Management; Quinolones; Respiratory Function Tests; Symptom Flare Up; Treatment Outcome | 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 |
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 |
Continuous glucose monitoring in youth with cystic fibrosis treated with lumacaftor-ivacaftor.
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Blood Glucose; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; DNA; Drug Combinations; Female; Forced Expiratory Volume; Glucose Tolerance Test; Glycated Hemoglobin; Homozygote; Humans; Male; Mutation; Prognosis; Quinolones; Treatment Outcome | 2019 |
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 |
Rate and predictors of prescription of lumacaftor - Ivacaftor in the 18 months following approval in the United States.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Chloride Channel Agonists; Cystic Fibrosis; Drug Combinations; Drug Prescriptions; Eligibility Determination; Female; Humans; Insurance Claim Review; Male; Product Surveillance, Postmarketing; Quinolones; Registries; Severity of Illness Index; United States | 2018 |
False-positive cannabinoid screens in adult cystic fibrosis patients treated with lumacaftor/ivacaftor.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cannabinoids; Chloride Channel Agonists; Cystic Fibrosis; Diagnostic Errors; Drug Combinations; False Positive Reactions; Female; Humans; Male; Middle Aged; Quinolones; Substance Abuse Detection | 2018 |
Cost-Effectiveness and Budget Impact of Lumacaftor/Ivacaftor in the Treatment of Cystic Fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Budgets; Clinical Decision-Making; Cost-Benefit Analysis; Cystic Fibrosis; Decision Support Techniques; Drug Combinations; Drug Costs; Forced Expiratory Volume; Humans; Lung; Models, Economic; Quality-Adjusted Life Years; Quinolones; Respiratory System Agents; Time Factors; 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 |
Tezacaftor/Ivacaftor (Symdeko) for cystic fibrosis.
Topics: Administration, Oral; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Administration Schedule; Drug Combinations; Drug Interactions; Humans; Indoles; Lung; Membrane Transport Modulators; Mutation; Quinolones; Treatment Outcome | 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 |
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have differential effects on cystic fibrosis macrophage function.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Case-Control Studies; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Cytokines; Drug Combinations; Female; Humans; Ion Transport; Macrophages; Male; Mutation; Pseudomonas aeruginosa; Pseudomonas Infections; Quinolones; Signal Transduction | 2018 |
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 |
Corticosteroid use and increased CXCR2 levels on leukocytes are associated with lumacaftor/ivacaftor discontinuation in cystic fibrosis patients homozygous for the F508del CFTR mutation.
Topics: Adrenal Cortex Hormones; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Forced Expiratory Volume; Gene Deletion; Homozygote; Humans; Leukocytes; Logistic Models; Male; Medication Adherence; Quinolones; Receptors, Interleukin-8B; Tetraspanin 30 | 2018 |
Predictive factors for lumacaftor/ivacaftor clinical response.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Biomarkers, Pharmacological; Child; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Monitoring; Female; Humans; Male; Mutation; Pharmacogenomic Testing; Quinolones; Respiratory Function Tests; Sweat; Treatment Outcome; Young Adult | 2019 |
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 |
Exhaled nitric oxide in stable adult cystic fibrosis patients, during exacerbation and following CFTR-modifying treatment.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Breath Tests; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Disease Progression; Drug Combinations; Exhalation; Female; Humans; Male; Nitric Oxide; Prospective Studies; Quinolones; Young Adult | 2019 |
Long-Term CFTR Modulators and Changes in Hemoglobin.
Topics: Aminophenols; Aminopyridines; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Hemoglobins; Humans | 2019 |
Effects of Lumacaftor/Ivacaftor on physical activity and exercise tolerance in three adults with cystic fibrosis.
Topics: Activities of Daily Living; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Monitoring; Exercise; Exercise Tolerance; Forced Expiratory Volume; Humans; Male; Middle Aged; Mutation; Oxygen Consumption; Quinolones; Treatment Outcome | 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 |
Impact of CFTR-modulating drugs on GH-IGF-1 axis impairment in adult patients with cystic fibrosis.
Topics: Adult; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Follow-Up Studies; Growth Disorders; Human Growth Hormone; Humans; Insulin-Like Growth Factor I; Male; Middle Aged; Mutation; Prognosis; Quinolones | 2019 |
Pathogen acquisition in patients with cystic fibrosis receiving ivacaftor or lumacaftor/ivacaftor.
Topics: Adolescent; Adult; Aminophenols; Aminopyridines; Benzodioxoles; Child; Child, Preschool; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Female; Humans; Male; Pseudomonas aeruginosa; Pseudomonas Infections; Quinolones; Retrospective Studies; Staphylococcal Infections; Staphylococcus aureus; Young Adult | 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 |
Lumacaftor/ Ivacaftor improves exercise tolerance in patients with Cystic Fibrosis and severe airflow obstruction.
Topics: Adult; Aminophenols; Aminopyridines; Australia; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Exercise Tolerance; Female; Genotype; Homozygote; Humans; Male; Middle Aged; Mutation; Quinolones; Respiratory Function Tests; Walk Test; Young Adult | 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 |
Pulmonology: CFTR modulators for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Humans; Lung; Mutation; Oxadiazoles; Quinolones | 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 |
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 |
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 |
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 |
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 |
Another Beginning for Cystic Fibrosis Therapy.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Male; Quinolones | 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 |
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 |
A new chapter in therapy for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Male; Quinolones | 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 |
Predicting CFTR activity with front-runner cystic fibrosis drugs.
Topics: Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Male; Mutation, Missense | 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 |
Precision Medicine: At What Price?
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cost-Benefit Analysis; Cystic Fibrosis; Drug Combinations; Humans; Precision Medicine; Quinolones; Respiratory System Agents; United States | 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 |
Orkambi's Slick Unveiling Puts Insurers in a Bind.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Drug Combinations; Drug Costs; Drug Industry; Humans; Insurance Coverage; Insurance, Pharmaceutical Services; Quinolones; United States | 2015 |
A combination therapy for cystic fibrosis.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Gene Deletion; History, 20th Century; History, 21st Century; Humans; Quinolones | 2015 |
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 |
Response to: 'Lumacaftor/ivacaftor for patients homozygous for Phe508del-CFTR: should we curb our enthusiasm?' by Jones and Barry.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Gene Deletion; Humans; Quinolones | 2016 |
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Male; Quinolones | 2015 |
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Male; Quinolones | 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 |
Game Changers.
Topics: Acute Disease; Aminophenols; Aminopyridines; Benzodioxoles; Brain Ischemia; Cystic Fibrosis; Drug Combinations; Endovascular Procedures; Humans; Quinolones; Rectal Neoplasms; Stroke; Transanal Endoscopic Microsurgery | 2015 |
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 |
Lumacaftor-ivacaftor (Orkambi) for cystic fibrosis: behind the 'breakthrough'.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Body Mass Index; Cystic Fibrosis; Data Interpretation, Statistical; Disease Progression; Drug Approval; Drug Combinations; Drug Costs; Evidence-Based Medicine; Humans; Lung; Quinolones; Surveys and Questionnaires; Treatment Outcome; United States; United States Food and Drug Administration | 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 |
[A milestone in cystic fibrosis therapy].
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Female; Humans; Male; Quinolones | 2015 |
[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 |
Lumacaftor/ivacaftor (Orkambi) for cystic fibrosis.
Topics: Administration, Oral; Aminophenols; Aminopyridines; Benzodioxoles; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Drug Costs; Drug Interactions; Humans; Membrane Transport Modulators; Mutation; Quinolones; Risk Factors; Treatment Outcome | 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 |
[Causal therapy is available].
Topics: Adolescent; Aminophenols; Aminopyridines; Benzodioxoles; Child; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; DNA Mutational Analysis; Drug Combinations; Humans; Quinolones | 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 |
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 |
Cystic Fibrosis: The Dawn of a New Therapeutic Era.
Topics: Aminophenols; Aminopyridines; Benzodioxoles; Chloride Channel Agonists; Cystic Fibrosis; Cystic Fibrosis Transmembrane Conductance Regulator; Drug Combinations; Early Diagnosis; Genetic Therapy; Humans; Quinolones; Survival Rate | 2017 |
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 |
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 |
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 |