October 31, 2019Open Access

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

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Abstract

Elexacaftor-tezacaftor-ivacaftor was efficacious in patients with cystic fibrosis with Phe508del-minimal function genotypes, in whom previous CFTR modulator regimens were ineffective. (Funded by Vertex Pharmaceuticals; VX17-445-102 ClinicalTrials.gov number, NCT03525444.).

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Authors

Peter G. Middleton

The University of Sydney

Marcus Mall

German Centre for Cardiovascular Research

Pavel Dřevı́nek

Charles University

Journals

New England Journal of Medicine

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Institutions

University of Washington

University of Toronto

KU Leuven

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Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

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Abstract

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Discussion

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