e-Article
Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials
Document Type
Article
Author
Uluer, Ahmet Z; MacGregor, Gordon; Azevedo, Pilar; Indihar, Veronica; Keating, Claire; Mall, Marcus A; McKone, Edward F; Ramsey, Bonnie W; Rowe, Steven M; Rubenstein, Ronald C; Taylor-Cousar, Jennifer L; Tullis, Elizabeth; Yonker, Lael M; Chu, Chenghao; Lam, Anna P; Nair, Nitin; Sosnay, Patrick R; Tian, Simon; Van Goor, Fredrick; Viswanathan, Lakshmi; Waltz, David; Wang, Linda T; Xi, Yingmei; Billings, Joanne; Horsley, Alexander; Horsley, Alexander; Nash, Edward F.; Bakker, Marleen; van der Meer, Renske; Merkus, Petrus; Majoor, Christof; McCoy, Karen; Billings, Joanne; Pancham, Krishna; Tolle, James; Quick, Bryon; Uluer, Ahmet; DiMango, Emily; Rao, Adupa; Reyes, Santiago; Klingsberg, Ross; Barreto, Celeste; Ortega, Victor; Willey-Courand, Donna; Schwarz, Carsten; Sutharsan, Sivagurunathan; Fischer, Rainald; Davies, Jane; Duckers, Jamie; Horsley, Alexander; Doe, Simon; Nash, Edward F.; Bakker, Marleen; Heijerman, Harry; van der Meer, Renske; Merkus, Petrus; Majoor, Christof; Solomon, George M.; Merlo, Christian; Griffonnet, Jennifer; Pilewski, Joseph; Dunitz, Jordan; Sheikh, Saba; Rubenstein, Ronald C.; Rosenbluth, Daniel B.; Liou, Theodore; Indihar, Maria; Pancham, Krishna; Yonker, Lael; Nasr, Samya; Griffonnet, Jennifer; Brown, Cynthia D.; Sawicki, Gregory S.; Ruddy, Jennifer; DiMango, Emily; Garcia, Bryan; Braun, Andrew; Gifford, Alex H.; Mehdi, Nighat; Tupayachi Ortiz, Maria; Jain, Raksha; Calimano, Francisco J.; Johannes, Jimmy; Daines, Cori L.; Fullmer, Jason; Mermis, Joel; Barrios, Christopher; Ly, Ngoc; Casserly, Brian P.; Eisenmann, Stephan; Hebestreit, Helge; Kiefer, Alexander; Sutharsan, Sivagurunathan; Fischer, Rainald; MacGregor, Gordon; Peckham, Daniel; Ledson, Martin; Van Braeckel, Eva; Merkus, Petrus; McElvaney, Noel Gerard; McKone, Edward; Plant, Barry; Burr, Lucy; Smith, Daniel J.; Middleton, Peter; Wilson, John
Source
The Lancet Respiratory Medicine; June 2023, Vol. 11 Issue: 6 p550-562, 13p
Subject
Language
ISSN
22132600; 22132619
Abstract
Elexacaftor–tezacaftor–ivacaftor has been shown to be safe and efficacious in people with cystic fibrosis and at least one F508delallele. Our aim was to identify a novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination capable of further increasing CFTR-mediated chloride transport, with the potential for once-daily dosing.