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

"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 ...

Cardiomyopathy is the leading cause of mortality in patients with DMD. Compared with their healthy counterparts, patients with DMD — including those with preserved left ventricular ejection fraction ...

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 ...

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

Capricor Therapeutics’ Duchenne muscular dystrophy therapy (DMD) appears to have slowed disease progression in older boys and young men who are in the later stages of their disease and unable to walk, ...

Investigators evaluated longitudinal MRI and spectroscopy outcomes and ambulatory function among 180 patients with Duchenne muscular dystrophy (DMD) to establish the utility and reproducibility of ...

A new surprise discovery could hold big implications for muscular dystrophy patients, with researchers finding that an existing cancer drug may delay progression of the hard-to-treat disease.

New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated. University of Rochester ...

Researchers at UBC’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy. Duchenne muscular dystrophy (DMD) is a ...