Viltolarsen targets dystrophin gene mutations, enabling functional dystrophin protein production, crucial for muscle health in patients with DMD. Consistent viltolarsen treatment slowed disease ...

Researchers at the University of South Florida USF Health Morsani College of Medicine have identified a potential ...

MILAN--(BUSINESS WIRE)--Italfarmaco Group announced today that the U.S. Food and Drug Administration (FDA) has completed its filing review and accepted the company’s New Drug Application (NDA) for ...

Researchers at the USF Health Morsani College of Medicine have identified a potential pathway that could protect cardiac ...

Capricor Therapeutics has presented positive long-term data from its HOPE-2 clinical trial at the 2025 Muscular Dystrophy Association Conference, indicating that its leading treatment, deramiocel, can ...

Delandistrogene moxeparvovec showed significant long-term stabilization or slowed progression in DMD patients over 3 and 5 years compared to external controls. Clinical trials demonstrated ...

Phase 3 EPIDYS MRI data quantify givinostat’s potential to preserve functional muscle tissue and reduce fat infiltration in ...

Duchenne muscular dystrophy (DMD) is a genetic disease that causes muscle weakness and motor skill difficulties due to the loss of muscle tissue from changes to dystrophin proteins. It mostly affects ...

"While we did see correlations between activity levels and cardiomyopathy progression in patients with Duchenne muscular dystrophy, these correlations were not particularly strong. Our interpretation ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

There is no cure for DMD, but treatments can slow the progression of the disease and help improve quality of life. Duchenne muscular dystrophy (DMD) is a chronic condition that causes a gradual loss ...