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Tag Archives: Duchenne

FDA rejects second Sarepta drug for Duchenne muscular dystrophy

S arepta Therapeutics suffered a surprise setback on Monday when the Food and Drug Administration rejected its request to commercialize a second medicine to treat children with Duchenne Muscular Dystrophy, a rare hereditary muscle wasting disease. [19659003] In a statement, Sarepta said the FDA has refused to approve its medicine called Vyondys 53 because of the risk of intravenous infusion …

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A race for a better Duchenne treatment

O [PfP] On Friday, Pfizer presented the first results for gene therapy for the treatment of human Duchenne muscular dystrophy. Pfizer shares remained unchanged, but Sarepta Therapeutics' shares, which developed its own treatment, rose 18%. The leading authors of STAT, Adam Feuerstein and Matthew Herper, have this morning with some strong caffeinated drinks together to discuss the results. Adam : …

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