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Scientists home in on cause of Duchenne gene therapy side effect

Duchenne muscular dystrophy, a degenerative disease mostly affecting young boys, is one of the top targets for gene therapy developers. Recently, however, the pace of …

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Sarepta, with new Duchenne results, to broach ‘possibility’ of speedy gene therapy approval

Dive Brief: Sarepta Therapeutics on Monday presented new results from a placebo-controlled study of its experimental gene therapy for Duchenne muscular dystrophy, a closely watched …

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