Panelists discuss how data from MDA 2025 demonstrate that delandistrogene moxeparvovec (gene therapy for DMD) shows statistically significant improvements in motor function outcomes including North ...

A retrospective cohort study found that, among 459 patients with DMD, constipation (32.5%) was the most common GI complication, followed by dysphagia (9.4%). Older age, lower body mass index, and use ...

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

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...

Genetic testing, often via a blood sample, can confirm a muscular dystrophy diagnosis. It can also identify specific gene mutations to help guide treatment and determine whether or not someone is a ...

The FDA has officially limited the label of Sarepta Therapeutics’ Elevidys, putting an end to a whirlwind few months that saw the abrupt departure—and reinstatement—of top agency official Vinay Prasad ...

Capricor Therapeutics’ cell therapy for Duchenne muscular dystrophy has come up short at the FDA, with the regulator rejecting the biotech’s application seeking regulatory approval for what the ...

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

Pathologic changes of the X-chromosome gene for dystrophin give rise to Duchenne muscular dystrophy (DMD), and dystrophin -- which minimizes muscle fiber loss due to sarcolemma contraction damage when ...

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 ...