FDA Grants Full Approval to Elevidys for Duchenne Muscular Dystrophy.

The article focuses on the FDA's granting of full approval to Elevidys, a gene therapy by Sarepta Therapeutics, for ambulatory patients aged 4 years and older with Duchenne muscular dystrophy, while also granting accelerated approval for non-ambulatory patients. Topics include the therapy's mechanism involving gene delivery for a functional form of dystrophin, its potential reach to a significant portion of patients with Duchenne.