VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

Keating, D.; Marigowda, Gautham; Burr, Lucy; Daines, Cori; Mall, Marcus; F. McKone, Edward; W. Ramsey, Bonnie; M. Rowe, Steven; A. Sass, Laura; Tullis, Elizabeth; McKee, Charlotte; M. Moskowitz, Samuel; Robertson, Sarah; R. K. Savage, J.; Simard, C; Van Goor, Fredrick; Waltz, David; Xuan, Fengjuan; Young, Timothy; L. Taylor‐Cousar, Jennifer

doi:10.1056/nejmoa1807120

Public

VX-445–Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis and One or Two Phe508del Alleles

Published in New England Journal of Medicine • Oct 18, 2018

Authors:

D. Keating

Gautham Marigowda

Lucy Burr

Abstract

BACKGROUND: VX-445 is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector designed to restore Phe508del CFTR protein function in patients with cystic fibrosis when administered with tezacaftor and ivacaftor (VX-445-tezacaftor-ivacaftor). METHODS: ) from baseline. R...

Subject

Ivacaftor

Cystic fibrosis

Cystic fibrosis transmembrane conductance regulator

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