U.S. Sen. Richard Blumenthal and researchers raise the alarm on how a federal medical research funding cap could impact "life ...

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

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

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

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

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

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

Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...

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

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

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

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