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The EMBARK Phase 3 study for Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, was published this month in Nature Medicine. The paper largely reiterates what’s already known about the negative study — Elevidys failed to achieve its primary endpoint of improving overall muscle function.
But when the paper discussed Elevidys’ impact on secondary muscle-function endpoints — the results that Sarepta touted as demonstrating the gene therapy’s benefit for patients — the EMBARK authors diverged from the company line.
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