A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

DMD is an X-linked disorder characterized by progressive muscle weakness and wasting due to the absence of a protein called dystrophin, which in turn causes degeneration of skeletal and cardiac muscle ...

Characterized by progressive muscle degeneration, this X-linked disorder results from mutations in the DMD gene, which encodes dystrophin, a protein essential for maintaining muscle cell integrity.

We know, for example, certain regions of the dystrophin gene may be more likely ... and therefore we're going to intervene with this muscle-supportive therapy at this moment, and we're going ...

Duchenne muscular dystrophy is a condition that primarily affects young boys, starving their bodies of dystrophin protein required for muscle function. The Food and Drug Administration has said ...

Elevidys was granted expanded approval by the U.S. Food and Drug Administration in 2024, with expectations high that the therapy, designed to restore a critical muscle protein called dystrophin ...