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Sarepta says patient dies after treatment with gene therapy

By Thomson Reuters Mar 18, 2025 | 7:17 AM

(Reuters) -Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for rare muscular dystrophy.

Shares of the company fell more than 25% to $75.86 in premarket trading.

The gene therapy, branded as Elevidys, is approved in the U.S. for patients with Duchenne muscular dystrophy aged four and older.

Acute liver injury is a known possible side effect of the therapy and mentioned in its prescribing information, the company said.

Although it is not a new safety signal, acute liver failure leading to death represents a severity of acute liver injury not previously reported for the therapy.

(Reporting by Sriparna Roy in Bengaluru; Editing by Shilpi Majumdar and Shinjini Ganguli)