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Update to the U.S. Duchenne Community on the Ataluren (Translarna) for Nonsense Mutation Duchenne Muscular Dystrophy
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Update to the U.S. Duchenne Community on the Ataluren (Translarna) for Nonsense Mutation Duchenne Muscular Dystrophy
Nonsense Mutation Duchenne Muscular DystrophyThe FDA is continuing its review of ataluren (Translarna), the first potential therapy specifically for individuals with nonsense mutation Duchenne (nmDMD). There is currently no set decision date. The application includes more than 10 years of safety and efficacy data.Read the letter to the community below:PTC Update to the Duchenne Community Aug 2025DownloadThe post Update to the U.S. Duchenne Commu…
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