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Article

VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

18 Oct 2018

Abstract excerpt

BACKGROUND: The next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector VX-659, in triple combination with tezacaftor and ivacaftor (VX-659-tezacaftor-ivacaftor), was developed to restore the function of Phe508del CFTR protein in patients with cystic fibrosis. METHODS: ). RESULTS: . The sweat chloride concentrations and scores on the respiratory domain of the Cystic Fibrosis...

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PMID: 30334693
DOI: 10.1056/nejmoa1807119

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VX-659–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del AllelesPMID 30334693Read ↗

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