Safety and efficacy of vanzacaftor–tezacaftor–deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials

Autor: 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
Zdroj: The Lancet Respiratory Medicine; June 2023, Vol. 11 Issue: 6 p550-562, 13p
Abstrakt: 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.
Databáze: Supplemental Index