A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation

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...

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Detalhes bibliográficos
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: 2011
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3230303/
https://ncbi.nlm.nih.gov/pubmed/22047557
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1105185
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