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First Duchenne gene therapy approved by FDA for young children

June 22, 2023

The Food and Drug Administration on Thursday approved the first gene therapy for Duchenne muscular dystrophy, a milestone in treatment for the deadly disease and a calculated gamble the medicine can slow its unrelenting progression.

The regulator granted the treatment, called Elevidys and developed by biotechnology company Sarepta Therapeutics, an “accelerated” clearance, meaning its benefit must be confirmed in further testing. Its use is limited to Duchenne patients who are 4 or 5 years of age, can still walk, and don’t have genetic mutations that might blunt the treatment’s effects or raise its safety risks.

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