A randomized placebo-controlled trial of ataluren for the treatment of nonsense mutation cystic fibrosis

BACKGROUND: Ataluren was developed to restore functional protein production in genetic disorders caused by nonsense mutations, which are the cause of cystic fibrosis (CF) in 10% of patients.. METHODS: This randomized, double-blind, placebo-controlled study enrolled 238 patients ≥6 years with nmCF to...

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Detaylı Bibliyografya
Asıl Yazarlar: EitanKerem, Konstan, Michael W., De Boeck, Kris, Accurso, Frank J., Sermet-Gaudelus, Isabelle, Wilschanski, Michael, Elborn, J S, Melotti, Paola, Bronsveld, Inez, Fajac, Isabelle, Malfroot, Anne, Rosenbluth, Daniel B., Walker, Patricia A., McColley, Susanna A., Knoop, Christiane, Quattrucci, Serena, Rietschel, Ernst, Zeitlin, Pamela L., Barth, Jay, Elfring, Gary L., Welch, Ellen M., Branstrom, Arthur, Spiegel, Robert J., Peltz, Stuart W., Ajayi, Temitayo, Rowe, Steven M.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2014
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Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC4154311/
https://ncbi.nlm.nih.gov/pubmed/24836205
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/S2213-2600(14)70100-6
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