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, would ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

The accelerated approval of Exondys 51 is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients. In this case the FDA said Sarepta ...

WVE-N531 showed significant improvements in functional measures and muscle health in DMD patients during the phase 2 FORWARD-53 trial. Sustained exon skipping, dystrophin restoration, and improved ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Biopsy data from eight of the boys showed a 6.4% mean muscle content-adjusted dystrophin. The average dystrophin between week 24 and 48 was 7.8%, the company said. Seven of these eight boys ...

It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm ...

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 ...