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Death of Elevidys Patient Rattles Duchenne Community

Summary by Managed Healthcare Executive
The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent cytomegalovirus infection.
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Managed Healthcare ExecutiveManaged Healthcare Executive

Death of Elevidys Patient Rattles Duchenne Community

The patient died of acute liver failure; the maker of the gene therapy noted that the patient also had a recent cytomegalovirus infection.

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contemporarypediatrics.comcontemporarypediatrics.com

Craig McDonald, MD, breaks down positive SGT-003 data for Duchenne muscular dystrophy

In this Q+A interview, Craig McDonald, MD, discusses the interim, 90-day data from the phase 1/2 INSPIRE DUCHENNE trial, evaluating SGT-003, a gene therapy candidate for DMD.

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Medical BriefMedical Brief

Young patient dies after new Duchenne gene therapy - Juta MedicalBrief

A young person with Duchenne muscular dystrophy died after treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said last week, adding that the patient had experienced acute liver failure. Testing showed he also had a recent cytomegalovirus (CMV) infection, which was a possible contributing factor, according to the treating physician. “Although it is not a new safety signal and the benef…

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Medical Brief broke the news in on Wednesday, March 26, 2025.
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