Translarna Increased Dystrophin Levels in Phase 2 DMD Study

Translarna Increased Dystrophin Levels in Phase 2 DMD Study

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Translarna Increased Dystrophin Levels in Phase 2 DMD Study

Treatment with Translarna (ataluren) increased levels of the protein dystrophin in young boys with Duchenne muscular dystrophy (DMD) caused by nonsense mutations, new clinical trial data show. The findings were presented in a webcast by Stuart W. Peltz, PhD, the CEO of PTC Therapeutics, which is developing Translarna. DMD is caused by a lack of the muscle protein dystrophin due to mutations in the DMD gene. Some people with DMD have a specific mutation called…

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