Researchers at Kumamoto University have demonstrated that iron supplementation can significantly alleviate muscle pathology ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. For ...

The FDA granted accelerated approval to delandistrogene moxeparvovec (Elevidys), the first gene therapy for Duchenne muscular dystrophy, the agency announced Thursday. The adeno-associated virus-based ...

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 ...