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 & ...

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 ...

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, ...

Interim clinical study results for the BB-301 Phase 1b/2a Treatment Study including 12-month follow-up results for the first four Cohort 1 completers, 24-month clinical study results for the first ...

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 ...

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 dystrophy affects approximately 1 in 3,500 male births. Though rare in females, there are documented cases. It is usually diagnosed between 3 and 6 years of age and is degenerative, causing ...

On March 10, 2026, from 12:00 p.m. -- 1:30 p.m. ET, in room Key West ABCD at the Hilton Orlando, Avidity will host an industry forum lunch titled "Biomarker Advancements in Rare Neuromuscular Disease: ...