• Patients treated with delandistrogene moxeparvovec at ages 8-12 demonstrated durable and clinically meaningful benefits across the North Star Ambulatory Assessment and timed function measures over 2 years compared to a well-matched external control group
  • Separate findings in children treated at ages 2 to under 3 demonstrated robust delandistrogene moxeparvovec-dystrophin expression and transduction efficiency at 12 weeks
  • In these analyses, the safety profile of delandistrogene moxeparvovec remained consistent and no new signals were identified

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data at the 31st Annual Congress of the World Muscle Society (WMS) that provide evidence of clinically meaningful functional benefit of ELEVIDYS (delandistrogene moxeparvovec) in older ambulatory patients with Duchenne muscular dystrophy.

In ambulatory patients treated at ages 8 to 12, an age range in which functional decline becomes even more pronounced, treatment was associated with durable benefits across multiple measures over two years compared to a well-matched external control. These findings suggest that the benefits previously observed in younger ambulatory patients are also being observed in older ambulatory patients. Sarepta also presented an analysis of patients treated at ages 2 and 3 that demonstrated robust delandistrogene moxeparvovec-dystrophin expression and sarcolemmal localization 12 weeks after infusion. Together, the analyses expand the evidence base across distinct ages and stages of ambulant Duchenne patients, while continued follow-up will be important to understand longer-term outcomes.