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A Duchenne Breakthrough Awaits In 2023 - But Risks Remain For Sarepta And US FDA

Gene Therapy Could Be Launched Mid-2023

Executive Summary

The FDA and Sarepta have had a controversial history with the accelerated approvals of the firm’s earlier drugs for Duchenne muscular dystrophy – but the company’s investigational gene therapy, SRP-9001, now under a priority review, could be a genuine breakthrough.

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