A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevydis, patient advocacy group Parent ...

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

Sarepta Therapeutics said that a young man died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy ...

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease----Data Presented at the 2025 Muscular ...

Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

Giulio Cossu, MD, speaks to the lingering safety concerns related to ex vivo gene therapy in Duchenne muscular dystrophy (DMD) as long-term data are yet to be established.

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The initial signs of Duchenne muscular dystrophy in a 3 or 4-year-old child may be subtle, explains Dr. Alexandra ...

Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. The young man died of acute ...