Article
Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation.
Chest - 1 Sept 2012
Flume Patrick A, Liou Theodore G, Borowitz Drucy S, Li Haihong, Yen Karl, Ordoñez Claudia L, Geller David E
Abstract excerpt
BACKGROUND: Ivacaftor (VX-770) is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that was approved in the United States for the treatment of cystic fibrosis (CF) in patients ≥ 6 years of age who have a G551D mutation; however, the most prevalent disease-causing CFTR mutation, F508del, causes a different functional defect. The objectives of this study were to evaluate the safety of...
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