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New orphan drug for Duchenne muscular dystrophy

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euEuropean Medicines Agency (EMA) grant conditional authorization of new orphan medicine intended for the treatment of Duchenne muscular dystrophy. Translarna (ataluren), will be available in the European Union after the final phase of clinical trials. The approval is based on results of safety and efficacy from a randomized, double-blind, multicenter study of 174 patients over 48 weeks. For more information, please visit the official website of EMA.

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