An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...

Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...

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, ...

Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.

Edgewise is sponsoring an Industry Forum, "Spotlight on Becker muscular dystrophy: Understanding the lived experience of Becker and clinical advancements with a novel agent." Only conference attendees ...

Edgewise Therapeutics' stock soars on positive Becker Muscular Dystrophy trial results. Learn why EWTX is a promising buy with derisked programs.

Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), with a group of more than 150 advocates, will head to Capitol Hill ...

Columnist Patrick Moeschen offers a retrospective of what he did and didn't know about living with muscular dystrophy over ...

Italfarmaco S.p.A. today announced a comprehensive update on the regulatory and clinical advancements for givinostat, the company’s drug for the treat ...