Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

Wainwright, Claire; Elborn, J.S.; W. Ramsey, Bonnie; Marigowda, Gautham; Huang, Xiaohong; Cipolli, Marco; Colombo, Carla; C. Davies, Jane; De Boeck, K.; A. Flume, Patrick; W. Konstan, Michael; A. McColley, Susanna; McCoy, Karen; F. McKone, Edward; Munck, À.; Ratjen, Félix; M. Rowe, Steven; Waltz, David; Boyle, Michael

doi:10.1056/nejmoa1409547

Public

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

Published in New England Journal of Medicine • May 17, 2015

Authors:

Claire Wainwright

J.S. Elborn

Bonnie W. Ramsey

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: through week 24 (calculated as a percentage) w...

Subject

Ivacaftor

Medicine

Cystic fibrosis

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