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Sarepta Therapeutics is one of the firms working on gene therapy and has enough evidence on hand for the potential treatment of Duchenne muscular dystrophy. The firm claims that it has enough clinical data that shows their treatment is safe and effective to treat muscle-wasting disorder. Though an important Phase 3 trial is still underway and may yield data the coming year, the firm is interacting with regulators for submission under approval pathways.

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