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 ...

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 ...

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

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

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

After bringing Zolgensma to market in 2019 as the first gene therapy for spinal muscular atrophy, Novartis is back with a ...

The East Stroudsburg University campus community is rallying behind a student’s effort to raise funding for muscular dystrophy that was inspired by her bond with a 9-year-old boy battling the ...

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, ...

Each shamrock sold for one dollar will help the Muscular Dystrophy Association help children and teens. "Their life span is to 16 years old, so they are struggling with this disease for so long ...

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

New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of ind ...