Article
A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.
The New England journal of medicine - 3 Nov 2011
Ramsey Bonnie W, Davies Jane, McElvaney N Gerard, Tullis Elizabeth, Bell Scott C, Dřevínek Pavel, Griese Matthias, McKone Edward F, Wainwright Claire E, Konstan Michael W, Moss Richard, Ratjen Felix, Sermet-Gaudelus Isabelle, Rowe Steven M, Dong Qunming, Rodriguez Sally, Yen Karl, Ordoñez Claudia, Elborn J Stuart
Abstract excerpt
BACKGROUND: Increasing the activity of defective cystic fibrosis transmembrane conductance regulator (CFTR) protein is a potential treatment for cystic fibrosis. METHODS: We conducted a randomized, double-blind, placebo-controlled trial to evaluate ivacaftor (VX-770), a CFTR potentiator, in subjects 12 years of age or older with cystic fibrosis and at least one G551D-CFTR mutation. Subjects were randomly assigned...
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