Sarepta Therapeutics Inc.

Duchenne muscular dystrophy is a major therapeutic focus, including studies in ambulatory and non-ambulatory populations and in patients with genotypes amenable to exon skipping approaches. The research portfolio includes work on eteplirsen, vesleteplirsen, and delandistrogene moxeparvovec for muscle function, dystrophin expression, and long-term safety.

• Duchenne muscular dystrophy (DMD)
• Exon 45 skipping and exon 53 skipping
• Dystrophin restoration
• Motor function and ambulation

Clinical activity also includes follow-up and treatment evaluation in patients with pre-existing AAV antibodies, reflecting interest in gene transfer therapy for broader DMD populations.