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Children's Hospital LA pauses use of Sarepta gene therapy for all patients
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Children's Hospital LA pauses use of Sarepta gene therapy for all patients
Jul 21, 2025 3:35 PM

July 21 (Reuters) - Children's Hospital Los Angeles,

citing recent U.S. Food and Drug Administration actions, on

Monday said it has paused usage of Sarepta Therapeutics' ( SRPT )

gene therapy Elevidys in all patients with muscular

dystrophy.

The FDA on Friday asked Sarepta to voluntarily halt

shipments of the gene therapy after a muscular dystrophy patient

who received a different, experimental treatment died. The

company said it did not plan to comply with the request and

would continue making the treatment available to ambulatory

patients.

The company in June suspended use of Elevidys for patients

whose disease had progressed to the point where they need a

wheelchair after two teenagers died from liver failure after

treatment.

Elevidys has full FDA approval for children with Duchenne

Muscular Dystrophy who are still able to walk, but was given

conditional approval for non-ambulatory patients.

Children's Hospital LA said its pause, effective July 18,

has been communicated to "affected patient families while it

awaits any further determination by the FDA."

The hospital is one of more than 70 authorized to administer

Elevidys. Five other major children's hospitals asked by Reuters

to comment on whether they planned to continue use of the gene

therapy did not immediately respond.

Shares of Sarepta, down about 90% year-to-date, fell 5% on

Monday to close at $13.32 on Nasdaq.

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