Gene therapy approaches include gene replacement, suppression, and editing, each matched to specific genetic mechanisms in hereditary hearing loss. Preclinical studies in rodent models show promise, ...

Sarepta Therapeutics said it has temporarily suspended shipments of Elevidys ® (delandistrogene moxeparvovec-rokl) for infusion in non-ambulatory patients following the second death in three months of ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.