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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Κύριοι συγγραφείς: 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.
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: 2014
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Article
Διαθέσιμο Online: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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