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

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 ...

Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...

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

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 ...

DMD affects approximately one in 3,600 male infants. DMD is a type of muscular dystrophy, a group of genetic diseases that cause muscles to weaken over time. DMD is one of the most severe forms of ...

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 ...

Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics ...

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