Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

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

doi:10.1056/nejmoa1709846

Public

Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del

Published in New England Journal of Medicine • Nov 3, 2017

Authors:

Jennifer L. Taylor‐Cousar

À. Munck

Edward F. McKone

Abstract

BACKGROUND: Combination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to target the underlying cause of disease in patients with cystic fibrosis. METHODS: In this phase 3, randomized, double-blind, mul...

Subject

Ivacaftor

Medicine

Cystic fibrosis

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