Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will present at the ...

Spinal muscular atrophy affects all the body's muscles. For a long time, it was considered a disease caused solely by the ...

A team of researchers has developed a wirelessly activated device that mimics the wavelike muscular function in the esophagus and small intestine responsible for transporting food and viscous fluids ...

For boys who start corticosteroids for DMD, being taller is tied with slower growth, and being older is tied to more weight ...

Please provide your email address to receive an email when new articles are posted on . Givinostat met the primary study endpoint of improved time to climb four stairs. Consistent results were ...

Please provide your email address to receive an email when new articles are posted on . Interim results also showed reduction of the neurodegenerative biomarker neurofilament light chain. Serious ...

ITF Therapeutics LLC, the U.S. affiliate of Italfarmaco, today announced that results on givinostat for the treatment of Duchenne muscular dystrophy (DMD) are being presented at the 2025 Neuromuscular ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.

A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in ...