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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Middleton et al. (Thu,) studied this question.

synapsesocial.com/papers/69d8ebf8d2f7327e70ae4811 https://doi.org/https://doi.org/10.1056/nejmoa1908639

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Journals

New England Journal of Medicine

Institutions

University of Washington

University of Toronto

KU Leuven

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