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

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

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

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

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...

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

Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...

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

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