Article
Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST).
The Lancet. Respiratory medicine - 1 Nov 2014
McKone Edward F, Borowitz Drucy, Drevinek Pavel, Griese Matthias, Konstan Michael W, Wainwright Claire, Ratjen Felix, Sermet-Gaudelus Isabelle, Plant Barry, Munck Anne, Jiang Ying, Gilmartin Geoffrey, Davies Jane C
Abstract excerpt
BACKGROUND: Ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, is approved for the treatment of patients with cystic fibrosis aged 6 years or older with Gly551Asp-CFTR. We assessed the safety and efficacy of ivacaftor during 96 weeks of PERSIST in patients with cystic fibrosis who completed a previous 48-week, placebo-controlled trial of the drug (STRIVE or ENVISION). METHODS: In...
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