Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology ...

Zeleciment basivarsen (z-basivarsen) demonstrated sustained functional improvement across multiple clinical measures in the ongoing ACHIEVE ...

There are celebrations galore as Jack Johnson – the inspiration for Wigan-based charity Joining Jack – marks his 18th ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology and functional decline in a mouse model of facioscapulohumeral muscular ...

Nov. 13 -- THURSDAY, Nov. 12 (HealthDay News) -- Researchers report that a drug used to treat pneumonia might serve as an effective treatment against a type of muscular dystrophy. They tested the drug ...

A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...

In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid ...

Forbes contributors publish independent expert analyses and insights. William A. Haseltine, Ph.D., covers genomics and regenerative medicine Today, the world stands at a crossroads in genetic medicine ...

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

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...