US EditionPatient dies following muscular dystrophy gene therapy, Sarepta reports
- A patient died from acute liver injury while receiving Sarepta Therapeutics' gene therapy, Elevidys, marking the therapy's first known patient death.
- The severity of this case was unprecedented, even though acute liver injury is a known side effect of the treatment.
- The U.S. FDA approved Elevidys in 2023, despite concerns about its effectiveness for Duchenne muscular dystrophy.
- Sarepta's shares dropped over 23% following the report of the death, highlighting investor reaction to the therapy's risks.
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Jackson Progress-Argus
Sarepta’s Gene Therapy Under Scrutiny After Patient Death
Key Takeaways
Patient Dies Following Gene Therapy Treatment for Duchenne Muscular Dystrophy
A patient with Duchenne muscular dystrophy has died following treatment with Elevidys, a gene therapy developed by Sarepta Therapeutics, the company announced in a safety update. The news sent the company’s stock down about 23 percent in morning trading on March 18. “We are profoundly saddened to share that a young man with Duchenne muscular dystrophy has passed away following treatment with ELEVIDYS, having suffered acute liver failure,” a comp…
MedPage Today
Anti-Aging, Acupuncture and Health NewsYoung Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
(MedPage Today) -- A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said in a statement Tuesday. The patient died after...
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