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Sarepta, gene therapy and FDA
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The European drug authority recommends refusal of marketing authorization after study shows no benefit over placebo.
Sarepta would need to conduct new analyses to validate the safety of Elevidys—which has had U.S. shipments paused by the ...
European regulators said that a Duchenne gene therapy should not be approved, imperiling the treatment that is also facing ...
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Investor's Business Daily on MSNThe Hits Keep Coming For Sarepta. Why Its Latest Setback Creates A 'Dangerous Precedent' For Gene Therapies.Sarepta stock plunged again Thursday on a report that the Food and Drug Administration will require additional clinical ...
The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.
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