{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T21:27:38Z","timestamp":1791235658966,"version":"4.1.0"},"reference-count":51,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2023,9,7]],"date-time":"2023-09-07T00:00:00Z","timestamp":1694044800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100014943","name":"Sarepta Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014943","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Annals of Neurology"],"published-print":{"date-parts":[[2023,11]]},"abstract":"<jats:sec><jats:title>Objective<\/jats:title><jats:p>Delandistrogene moxeparvovec is approved in the USA for the treatment of ambulatory patients (4\u20135\u2009years) with Duchenne muscular dystrophy. ENDEAVOR (SRP\u20109001\u2010103; NCT04626674) is a single\u2010arm, open\u2010label study to evaluate delandistrogene moxeparvovec micro\u2010dystrophin expression, safety, and functional outcomes following administration of commercial process delandistrogene moxeparvovec.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>In cohort 1 of ENDEAVOR (N\u2009=\u200920), eligible ambulatory males, aged \u22654 to &lt;8\u2009years, received a single intravenous infusion of delandistrogene moxeparvovec (1.33\u2009\u00d7\u200910<jats:sup>14<\/jats:sup>\u2009vg\/kg). The primary endpoint was change from baseline (CFBL) to week\u200912 in delandistrogene moxeparvovec micro\u2010dystrophin by western blot. Additional endpoints evaluated included: safety; vector genome copies; CFBL to week\u200912 in muscle fiber\u2010localized micro\u2010dystrophin by immunofluorescence; and functional assessments, including North Star Ambulatory Assessment, with comparison with a propensity score\u2010weighted external natural history control.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The 1\u2010year safety profile of commercial process delandistrogene moxeparvovec in ENDEAVOR was consistent with safety data reported in other delandistrogene moxeparvovec trials (NCT03375164 and NCT03769116). Delandistrogene moxeparvovec micro\u2010dystrophin expression was robust, with sarcolemmal localization at week\u200912; mean (SD) CFBL in western blot, 54.2% (42.6); <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001. At 1\u2009year, patients demonstrated stabilized or improved North Star Ambulatory Assessment total scores; mean (SD) CFBL, +4.0 (3.5). Treatment versus a propensity score\u2010weighted external natural history control demonstrated a statistically significant difference in least squares mean (standard error) CFBL in North Star Ambulatory Assessment, +3.2 (0.6) points; <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>Results confirm efficient transduction of muscle by delandistrogene moxeparvovec. One\u2010year post\u2010treatment, delandistrogene moxeparvovec was well tolerated, and demonstrated stabilized or improved motor function, suggesting a clinical benefit for patients with Duchenne muscular dystrophy. 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