Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

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 mutatio...

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Главные авторы: Flume, Patrick A., Liou, Theodore G., Borowitz, Drucy S., Li, Haihong, Yen, Karl, Ordoñez, Claudia L., Geller, David E.
Формат: Artigo
Язык:Inglês
Опубликовано: American College of Chest Physicians 2012
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Original Research
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC3435140/
https://ncbi.nlm.nih.gov/pubmed/22383668
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1378/chest.11-2672
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