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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Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
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Article
Acesso em linha: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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