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FDA Rejects PTC’s New Drug Application for Translarna as a Nonsense Mutation DMD Treatment – Muscular Dystrophy News

Friday, 26 February, 2016

FDA Rejects PTC’s New Drug Application for Translarna as a Nonsense Mutation DMD Treatment – Muscular Dystrophy News

Ataluren is a small-molecule protein restoration therapy compound in clinical development for the treatment of Duchenne muscular dystrophy (DMD) caused by a nonsense mutation (nmDMD), cystic fibrosis caused by a nonsense mutation (nmCF), and other ...

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Source:FDA Rejects PTC's New Drug Application for Translarna as a Nonsense Mutation DMD Treatment - Muscular Dystrophy News

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