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Sarepta reports second case of liver failure death after its gene therapy treatment
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Sarepta reports second case of liver failure death after its gene therapy treatment
Jun 14, 2025 11:36 PM

June 15 (Reuters) - Sarepta Therapeutics ( SRPT ) on

Sunday said there had been a second reported case of acute liver

failure resulting in death after a patient received the

company's gene therapy for a rare form of muscular dystrophy.

The patient was undergoing treatment with Sarepta's therapy

called Elevidys, which is the only gene therapy approved by the

U.S. Food and Drug Administration for Duchenne muscular

dystrophy patients aged four and above.

The first instance of death was reported in March this year,

a 16-year-old boy who died from acute liver failure months after

receiving Elevidys.

Liver damage is a known risk with Elevidys and other gene

therapies that use adeno-associated viral vectors to infuse

modified genes.

Sarepta said it is taking steps to mitigate the risk of

acute liver failure in patients by working to convene an

independent group of experts in Duchenne and liver health to

consider an enhanced immunosuppression regimen for Elevidys. It

has also temporarily suspending shipments of the therapy and

informed the FDA and global health authorities about the

incident.

Roche, which partners with Sarepta for

commercialization of the gene therapy outside the United States,

said in a separate statement on Sunday that it has paused the

dosing of Elevidys in patients following the two cases of fatal

acute liver failure.

(Reporting by Rishabh Jaiswal in Bengaluru; Editing by Lincoln

Feast.)

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