The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...

Nov 3 (Reuters) - Sarepta (SRPT.O), opens new tab said on Monday that its late-stage study testing two gene-targeted therapies for Duchenne muscular dystrophy did not meet the main goal, sending its ...

Muscle weakness in Duchenne muscular dystrophy can lead to symptoms related to mobility, coordination, posture, and breathing, as well as other health effects. Duchenne muscular dystrophy (DMD) is a ...

Duchenne muscular dystrophy (DMD) causes muscle weakness that becomes more severe over time. While there is no cure, treatments can help slow the progression and manage symptoms and complications.

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 ...

Please provide your email address to receive an email when new articles are posted on . Cardiomyopathy in Duchenne muscular dystrophy possesses a unique pathophysiology. Integrated wall stress can be ...

At the FDA's request, delandistrogene moxeparvovec (Elevidys), the only approved gene therapy for Duchenne muscular dystrophy, will carry a black box warning for acute liver injury and acute liver ...

Physical activity and rehabilitation are key components of Duchenne muscular dystrophy treatment, despite recent drug therapy advances. "Rehabilitation care and rehabilitation teams with experience ...

This story is republished from STAT, the health and medicine news site that’s a partner to the Globe. Sign up for STAT’s free Morning Rounds newsletter here. Cambridge-based Sarepta Therapeutics said ...

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 ...

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 ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...