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

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

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

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...

FDA has requested the HOPE-3 clinical study report (CSR) as part of the BLA review processCompany expects to submit updates to the BLA in ...

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 ...

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

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

"High clinical suspicion is important for this population of patients. Since muscular dystrophy is progressive in nature, monitoring disease progression in these patients is critical for timing ...

Muscle tissue damage appears when muscular dystrophy is induced in a mouse model (middle). But when researchers block the function of two genes that drive unwanted mitochondrial pore formation (right) ...

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 ...

Tributes flooded the internet for Gilbert Gottfried after his family shared on Tuesday via Twitter that he died following “a long illness.” Since then, there has been more awareness around muscular ...