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- 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<\/i><\/b><\/li>\n<\/ul>\n
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- Separate findings in children treated at ages 2 to under 3 demonstrated robust delandistrogene moxeparvovec-dystrophin expression and transduction efficiency at 12 weeks<\/i><\/b><\/li>\n<\/ul>\n
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- In these analyses, the safety profile of delandistrogene moxeparvovec remained consistent and no new signals were identified<\/i><\/b><\/li>\n<\/ul>\n
CAMBRIDGE, Mass.–(BUSINESS WIRE<\/a>)–
\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today presented new data at the 31st<\/sup> 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.<\/p>\n\nIn 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.<\/p>\n
\n\u201cThe period between 8 and 12 years of age represents a critical phase of Duchenne when many boys transition from relative stability to progressive functional decline,\u201d said Louise Rodino-Klapac, Ph.D., president, Research & Development and Technical Operations, Sarepta. \u201cResults presented at WMS 2026 fit within a broader and increasingly consistent body of evidence generated across the clinical development program. Seeing similar patterns emerge across ambulatory Duchenne populations reinforce that timely treatment with delandistrogene moxeparvovec has the potential to meaningfully change the trajectory of the disease. We remain committed to following patients over time and publishing evidence to help inform treatment decisions and advance care for the Duchenne community.\u201d<\/p>\n
\n\u201cThe older ambulatory patient data demonstrate that delandistrogene moxeparvovec treatment can provide functional benefit that\u2019s statistically significant at a stage when decline often becomes more pronounced,\u201d said Craig McDonald, M.D., professor and chair of the UC Davis Health Department of Physical Medicine and Rehabilitation, an investigator in the EMBARK and ENDEAVOR studies and an author on both presentations. \u201cIn addition, the findings in younger children help us better understand the biological effects of treatment earlier in the disease course where preserving muscle may offer the greater opportunity for long-term benefit. While measures of expression provide important biological evidence, long-term functional outcomes remain the most meaningful way to understand the impact of treatment for patients and families.\u201d<\/p>\n
\nDetails from the delandistrogene moxeparvovec analyses:<\/p>\n
1.103LBP: Functional and safety outcomes in older ambulatory patients with Duchenne muscular dystrophy treated with delandistrogene moxeparvovec (C. McDonald, et al.<\/i>)<\/b><\/p>\n
\nThis analysis used a prospectively specified approach to compare treated patients with similar patients from several well-characterized DMD natural history datasets. Patients were matched based on the study eligibility criteria, and the groups were further balanced for key factors that can influence disease progression, with the aims of creating a reliable comparison and addressing common limitations of external control (EC) analyses.<\/p>\n
\nPatients from the EMBARK and ENDEAVOR studies who were aged 8-12 years at time of dosing (n=25), were compared to a well-matched EC group (n=99). Treatment with delandistrogene moxeparvovec demonstrated durable and clinically meaningful functional benefits over two years compared to EC. Results on the North Star Ambulatory Assessment (NSAA) and timed function tests as follows:<\/p>\n
- In these analyses, the safety profile of delandistrogene moxeparvovec remained consistent and no new signals were identified<\/i><\/b><\/li>\n<\/ul>\n
- Separate findings in children treated at ages 2 to under 3 demonstrated robust delandistrogene moxeparvovec-dystrophin expression and transduction efficiency at 12 weeks<\/i><\/b><\/li>\n<\/ul>\n
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