RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

Ifetroban demonstrated significant 5.4% improvement in cardiac functionNASHVILLE, Tenn., March 19, 2025 /PRNewswire/ -- Cumberland Pharma ...

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

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

On Monday, 17 March, the Muscular Dystrophy UK President’s Awards took place, in association with Airnow Technology and ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...

On Monday 17 March, the Muscular Dystrophy UK President’s Awards took place, in association with Airnow Technology and hosted ...

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

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, ...

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

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 ...