New Duchenne Therapy Shows Signs Of Reversing Muscle Loss

Duchenne therapy may be evolving from slowing disease to reversing it—with RNA approaches offering the prospect of restoring function. Ongoing research will determine if these advances can deliver lasting, transformative recovery. Image by freepik

New clinical evidence points to a potential breakthrough: a therapy that may not just slow but actually reverse muscle loss in boys with Duchenne muscular dystrophy. While existing treatments, such as corticosteroids and gene-targeted drugs, only modestly delay symptoms, the new findings—if validated in larger studies—could mark a shift from managing decline to possibly regaining lost muscle function. This would offer new hope in a previously incurable and relentlessly progressive disease.

A New Approach With RNA

Sarepta Therapeutics cur