Article
Tezacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del.
The New England journal of medicine - 23 Nov 2017
Taylor-Cousar Jennifer L, Munck Anne, McKone Edward F, van der Ent Cornelis K, Moeller Alexander, Simard Christopher, Wang Linda T, Ingenito Edward P, McKee Charlotte, Lu Yimeng, Lekstrom-Himes Julie, Elborn J Stuart
Abstract excerpt
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, multicenter, placebo-controlled, parallel-group trial, we evaluated combination therapy with tezacaftor and ivacaftor...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
