Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for F508del-CFTR and a gating or residual function mutation

Autor: Chmiel, J, Barry, Peter J, Colombo, Carla, De Wachter, Elke, Fajac, Isabelle, Mall, Marcus A, McBennett, Kimberly A, McKone, Edward F, Mondejar-Lopez, Pedro, Quon, Bradley, Ramsey, Bonnie W, Robinson, Padraic Sean, Sutharsan, S, Ahluwalia, Neil, Lu, M, Moskowitz, Samuel M, Prieto-Centurion, Valentin, Tian, Simon, Waltz, David, Weinstock, T, Xuan, Fengjuan, Zelazoski, L, Y, Zhang, Polineni, Deepika

Background: The triple combination regimen of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be safe and efficacious in people with CF aged ≥ 12 years with cystic fibrosis (CF) and heterozygous for F508del-CFTR and either a CFTR gating

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______3848::2caa15dd808d1be1d30689f429dd3719
https://doi.org/10.1016/s1569-1993(22)00875-x

163 Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children 6 years and older with cystic fibrosis and at least one F508del alleles: 96-week interim results from an open-label extension study.

Autor: Wainwright, C. (AUTHOR), McColley, S. (AUTHOR), McNally, P. (AUTHOR), Powers, M. (AUTHOR), Ratjen, F. (AUTHOR), Rayment, J. (AUTHOR), Retsch-Bogart, G. (AUTHOR), Roesch, E. (AUTHOR), Ahluwalia, N. (AUTHOR), Chin, A. (AUTHOR), Chu, C. (AUTHOR), Lu, M. (AUTHOR), Menon, P. (AUTHOR), Moskowitz, S. (AUTHOR), Waltz, D. (AUTHOR), Weinstock, T. (AUTHOR), Xuan, F. (AUTHOR), Zelazoski, L. (AUTHOR), Davies, J. (AUTHOR)

Publikováno v: Journal of Cystic Fibrosis. 2022 Supplement 2, Vol. 21, pS96-S96. 1p.