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

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

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

While plenty of people are going green in honor of Saint Patrick's Day, one student at East Stroudsburg University is turning ...

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 Bonner of the Center for Pediatric Neurosciences at ...

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.

Duchenne muscular dystrophy (DMD) is a genetic condition ... Each time a person who is a carrier of DMD has a child who is assigned male at birth, the child has a 50% chance of developing DMD.

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

there are rare cases of spontaneous mutations of the DMD in children whose birth mothers are not carriers of the condition. How Is Duchenne Muscular Dystrophy Diagnosed? DMD is diagnosed through a ...

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

REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated ...