Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial
(2021)
Journal Article
Sutharsan, S., McKone, E. F., Downey, D. G., Duckers, J., MacGregor, G., Tullis, E., Van Braeckel, E., Wainwright, C. E., Watson, D., Ahluwalia, N., Bruinsma, B. G., Harris, C., Lam, A. P., Lou, Y., Moskowitz, S. M., Tian, S., Yuan, J., Waltz, D., Mall, M. A., Aurora, P., …Wark, P. (2022). Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial. Lancet Respiratory Medicine, 10(3), 267-277. https://doi.org/10.1016/S2213-2600%2821%2900454-9
Background: Elexacaftor plus tezacaftor plus ivacaftor is a triple-combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator regimen shown to be generally safe and efficacious in people with cystic fibrosis aged 12 years or old...
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