Article
Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.
The New England journal of medicine - 16 Jul 2015
Wainwright Claire E, Elborn J Stuart, Ramsey Bonnie W, Marigowda Gautham, Huang Xiaohong, Cipolli Marco, Colombo Carla, Davies Jane C, De Boeck Kris, Flume Patrick A, Konstan Michael W, McColley Susanna A, McCoy Karen, McKone Edward F, Munck Anne, Ratjen Felix, Rowe Steven M, Waltz David, Boyle Michael P
Abstract excerpt
BACKGROUND: Cystic fibrosis is a life-limiting disease that is caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is the most common CFTR mutation. METHODS: We conducted two phase 3, randomized, double-blind, placebo-controlled studies that were designed to assess the effects of lumacaftor (VX-809), a CFTR corrector, in combination with...
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