Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

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Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

Taylor-Cousar, Jennifer L; Munck, Anne; McKone, Edward F; van der Ent, Cornelis K; Moeller, Alexander; Simard, Christopher; Wang, Linda T; Ingenito, Edward P; McKee, Charlotte; Lu, Yimeng; Lekstrom-Himes, Julie; Elborn, J. Stuart

The New England journal of medicine, 11/2017, Volume: 377, Issue: 21

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Patients with homozygous Phe508del cystic fibrosis were assigned to receive combination tezacaftor–ivacaftor or placebo for 24 weeks. The combination resulted in an FEV 1 that was 4 percentage points higher and a pulmonary-exacerbation rate that was 35% lower than with placebo.

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