Article
Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Dec 2014
De Boeck Kris, Munck Anne, Walker Seth, Faro Albert, Hiatt Peter, Gilmartin Geoffrey, Higgins Mark
Abstract excerpt
BACKGROUND: Ivacaftor is used to treat patients with CF and a G551D gating mutation; the KONNECTION study assessed the efficacy and safety of ivacaftor in patients with CF and a non-G551D gating mutation. METHODS: Patients with CF ≥6-years- old with non-G551D gating mutations received ivacaftor 150mg q12h or placebo for 8weeks in this 2-part, double-blind crossover study (Part 1) with a 16-week open-label...
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