Article
Lumacaftor/ivacaftor in people with cystic fibrosis with an A455E-CFTR mutation.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Sept 2021
Berkers Gitte, van der Meer Renske, Heijerman Harry, Beekman Jeffrey M, Boj Sylvia F, Vries Robert G J, van Mourik Peter, Doyle Jamie R, Audhya Paul, Yuan Zheng Jason, Kinnman Nils, van der Ent C Kors
Abstract excerpt
BACKGROUND: Previous in vitro organoid data showed A455E-CFTR, a rare CFTR mutation with 4.1% prevalence in the Netherlands, responds to lumacaftor/ivacaftor (LUM/IVA). We explored LUM/IVA's clinical efficacy in people with CF and ≥1 A455E-CFTR mutation. METHODS: Participants aged ≥12 years were randomized to 1 of 2 treatment sequences (LUM/IVA→placebo or placebo→LUM/IVA) with an 8-week washout period between....
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