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 ...

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

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

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 ...

Regenxbio has reported positive interim data from the Phase I/II portion of its Affinity Duchenne study, which is examining ...

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

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the “severity” of the patient’s case had not previously been seen with the therapy, ...

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 ...

"He was diagnosed with Duchenne Muscular Dystrophy when ... and I found out that insurance denies a quarter of every child with Muscular Dystrophy of mobility aids," explained Taylor.