Article
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.
The Lancet. Respiratory medicine - 1 Mar 2022
Sutharsan Sivagurunathan, McKone Edward F, Downey Damian G, Duckers Jamie, MacGregor Gordon, Tullis Elizabeth, Van Braeckel Eva, Wainwright Claire E, Watson Danie, Ahluwalia Neil, Bruinsma Bote G, Harris Christopher, Lam Anna P, Lou Yiyue, Moskowitz Samuel M, Tian Simon, Yuan Jason, Waltz David, Mall Marcus A
Abstract excerpt
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 older with at least one F508del-CFTR allele. We aimed to assess the magnitude and durability of the clinical effects of this triple combination regimen in people with...
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