Long-term data from DEVOTE/ONWARD studies show benefits of high dose nusinersen in people living with spinal muscular atrophy (SMA)New Phase 1b ...

The MDA meeting is a key opportunity to highlight the expanding real‑world evidence shaping care for the Duchenne community,” said Will Andrews, MD, Chief Medical Officer of Catalyst. “Through our ...

A RECOMMENDATION for the HSE not to cover the cost of a drug to help children with a rare muscular disease has been blasted by families. Last year, The Irish Sun highlighted the plight of ...

Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...

There are more than 30 different kinds of muscular dystrophy (MD), which are muscle diseases that are caused by mutations (or variations) in a person’s genes. The abnormal genes disrupt the production ...

REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its RGX-202 investigational gene therapy for Duchenne muscular dystrophy at the 2026 Muscular Dystrophy Association (MDA) Clinical & ...

When you think about children diagnosed with Duchenne and Becker muscular dystrophies, the first things that usually come to mind are difficulty walking and the progressive skeletal muscle wasting, ...

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

Muscular Dystrophies are a group of genetic conditions characterized by muscle weaknesses. They are genetic conditions and have different sub-categories depending on which area of muscle mass is ...