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Study on Sarepta’s Duchenne therapy diverges from the company line

October 24, 2024
in Health
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This story first appeared in Adam’s Biotech Scorecard, a subscriber-only e-newsletter. STAT+ subscribers can join right here to get it delivered to their inbox.

The EMBARK Section 3 examine for Elevidys, Sarepta Therapeutics’ gene remedy for Duchenne muscular dystrophy, was printed this month in Nature Medication. The paper largely reiterates what’s already recognized concerning the damaging examine — Elevidys failed to attain its main endpoint of bettering total muscle operate.

However when the paper mentioned Elevidys’ affect on secondary muscle-function endpoints — the outcomes that Sarepta touted as demonstrating the gene remedy’s profit for sufferers — the EMBARK authors diverged from the corporate line.

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