{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T19:12:42Z","timestamp":1792177962126,"version":"4.4.0"},"reference-count":16,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2026,6,3]],"date-time":"2026-06-03T00:00:00Z","timestamp":1780444800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,6,3]],"date-time":"2026-06-03T00:00:00Z","timestamp":1780444800000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/100014943","name":"Sarepta Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014943","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005693","name":"Parent Project Muscular Dystrophy","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005693","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2026,8]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Aims<\/jats:title>\n                    <jats:p>We report 5\u2010year results from a phase 1\/2a study of delandistrogene moxeparvovec, a recombinant adeno\u2010associated virus serotype rh74 vector\u2010based gene therapy for Duchenne muscular dystrophy (DMD), with post hoc analyses contextualizing functional outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      Four ambulatory patients with DMD (\u2265\u20094\u2013&lt;\u20098\u2009years at enrollment) entered an open\u2010label trial (Study 101; NCT03375164), receiving a single intravenous dose of delandistrogene moxeparvovec (2.0\u2009\u00d7\u200910\n                      <jats:sup>14<\/jats:sup>\n                      vg\/kg by supercoiled quantitative polymerase chain reaction (qPCR), equivalent to 1.33\u2009\u00d7\u200910\n                      <jats:sup>14<\/jats:sup>\n                      vg\/kg by linear qPCR). Safety and functional outcomes (including North Star Ambulatory Assessment [NSAA] total score, time to rise [TTR] from floor, 10\u2010m walk\/run [10MWR] time) were assessed. In post hoc analyses, functional outcomes of delandistrogene moxeparvovec\u2013treated patients were compared with propensity\u2010score\u2010weighted external controls (ECs) and natural history predictions.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      No new safety signals were reported 5\u2009years post\u2010infusion. One patient had cardiomyopathy at year 5; however, this event was deemed unrelated to treatment. All delandistrogene moxeparvovec\u2013treated patients (mean age, 10.2\u2009years) remained ambulant. The 5\u2010year NSAA total score mean change from baseline (standard deviation) for treated patients versus ECs was +7.5 (2.4) versus \u22123.9 (2.9) (least\u2010squares mean between\u2010group difference [standard error]: 9.8 [3.5],\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.0127). TTR and 10MWR mean times were stable, representing clinically meaningful differences versus ECs. An increased divergence in NSAA total score from 5\u2010year natural history predictions favoring gene therapy was also seen.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Findings support the long\u2010term, manageable safety profile of delandistrogene moxeparvovec in ambulatory patients with appropriate monitoring and demonstrate stabilization or delayed disease progression with treatment compared with matched untreated ECs.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70276","type":"journal-article","created":{"date-parts":[[2026,6,4]],"date-time":"2026-06-04T05:00:27Z","timestamp":1780549227000},"page":"397-403","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Five\u2010Year Outcomes With Delandistrogene Moxeparvovec in Patients With Duchenne Muscular Dystrophy: A Phase 1\/2a Study"],"prefix":"10.1002","volume":"74","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2691-0200","authenticated-orcid":false,"given":"Jerry R.","family":"Mendell","sequence":"first","affiliation":[{"name":"Jerry Mendell Center for Gene Therapy, Nationwide Children's Hospital  Columbus Ohio USA"},{"name":"Department of Pediatrics The Ohio State University  Columbus Ohio USA"},{"name":"Sarepta Therapeutics Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2974-3943","authenticated-orcid":false,"given":"Zarife","family":"Sahenk","sequence":"additional","affiliation":[{"name":"Jerry Mendell Center for Gene Therapy, Nationwide Children's Hospital  Columbus Ohio USA"},{"name":"Department of Pediatrics The Ohio State University  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4206-0557","authenticated-orcid":false,"given":"Linda P.","family":"Lowes","sequence":"additional","affiliation":[{"name":"Jerry Mendell Center for Gene Therapy, Nationwide Children's Hospital  Columbus Ohio USA"},{"name":"Department of Pediatrics The Ohio State University  Columbus Ohio USA"},{"name":"Sarepta Therapeutics Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2800-3855","authenticated-orcid":false,"given":"Megan A.","family":"Iammarino","sequence":"additional","affiliation":[{"name":"Jerry Mendell Center for Gene Therapy, Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2263-7569","authenticated-orcid":false,"given":"Lindsay N.","family":"Alfano","sequence":"additional","affiliation":[{"name":"Jerry Mendell Center for Gene Therapy, Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8779-3220","authenticated-orcid":false,"given":"Craig M.","family":"McDonald","sequence":"additional","affiliation":[{"name":"Departments of Physical Medicine &amp; Rehabilitation and Pediatrics University of California Davis Health  Sacramento California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4067-8962","authenticated-orcid":false,"given":"James E.","family":"Signorovitch","sequence":"additional","affiliation":[{"name":"Analysis Group Inc  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jim","family":"Jin","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing","family":"Li","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7649-9026","authenticated-orcid":false,"given":"Stefanie","family":"Mason","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8966-5177","authenticated-orcid":false,"given":"Louise R.","family":"Rodino\u2010Klapac","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,6,3]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.05.016"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0221097"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1080\/14712598.2020.1725469"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.1484"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27955"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41591-024-03304-z"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.3389\/fcell.2023.1167762"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.26755"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120\u2010025\u201000879\u20108"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2022.4315"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/dmcn.15176"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0304099"},{"key":"e_1_2_12_16_1","unstructured":"Sarepta Therapeutics Inc. \u201cELEVIDYS Prescribing Information \u201d(2025) https:\/\/www.elevidys.com\/pi."},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2022.02.009"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70276","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.70276","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.70276","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,9,11]],"date-time":"2026-09-11T22:05:02Z","timestamp":1789164302000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.70276"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,6,3]]},"references-count":16,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2026,8]]}},"alternative-id":["10.1002\/mus.70276"],"URL":"https:\/\/doi.org\/10.1002\/mus.70276","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,6,3]]},"assertion":[{"value":"2025-07-25","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-05-04","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-06-03","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}