{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T13:03:55Z","timestamp":1791378235501,"version":"4.3.2"},"reference-count":11,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2023,8,14]],"date-time":"2023-08-14T00:00:00Z","timestamp":1691971200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100007520","name":"Nationwide Children's Hospital","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007520","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"}]},{"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":["Muscle and Nerve"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>\n                      Delandistrogene moxeparvovec is indicated in the United States for the treatment of ambulatory pediatric patients aged 4 through 5\u2009years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the\n                      <jats:italic>DMD<\/jats:italic>\n                      gene. Long\u2010term delandistrogene moxeparvovec microdystrophin protein (a shortened dystrophin that retains key functional domains of the wild\u2010type protein) expression may positively alter disease progression in patients with DMD. We evaluated long\u2010term safety and functional outcomes of delandistrogene moxeparvovec in patients with DMD.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      An open\u2010label, phase 1\/2a, nonrandomized controlled trial (Study 101; NCT03375164) enrolled ambulatory males, \u22654 to &lt;8\u2009years old, with DMD. Patients received a single intravenous infusion (2.0\u2009\u00d7\u200910\n                      <jats:sup>14<\/jats:sup>\n                      vg\/kg by supercoiled quantitative polymerase chain reaction) of delandistrogene moxeparvovec and prednisone (1\u2009mg\/kg\/day) 1\u2009day before to 30\u2009days after treatment. The primary endpoint was safety. Functional outcomes were change from baseline in North Star Ambulatory Assessment (NSAA) and timed function tests.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Four patients (mean age, 5.1\u2009years) were enrolled. There were 18 treatment\u2010related adverse events; all occurred within 70\u2009days posttreatment and resolved. Mean NSAA total score increased from 20.5 to 27.5, baseline to year 4, with a mean (standard deviation) change of +7.0 (2.9). Post hoc analysis demonstrated a statistically significant and clinically meaningful 9\u2010point difference in NSAA score, relative to a propensity\u2010score\u2013weighted external control cohort (least\u2010squares mean [standard error]\u2009=\u20099.4 [3.4];\n                      <jats:italic>P<\/jats:italic>\n                      \u2009=\u2009.0125).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Gene transfer therapy with delandistrogene moxeparvovec treatment is well tolerated, with a favorable safety profile. Functional improvements are sustained through 4\u2009years, suggesting delandistrogene moxeparvovec may positively alter disease progression.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.27955","type":"journal-article","created":{"date-parts":[[2023,8,14]],"date-time":"2023-08-14T04:29:46Z","timestamp":1691987386000},"page":"93-98","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":85,"title":["Long\u2010term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1\/2a nonrandomized trial"],"prefix":"10.1002","volume":"69","author":[{"given":"Jerry R.","family":"Mendell","sequence":"first","affiliation":[{"name":"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"}]},{"given":"Zarife","family":"Sahenk","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy Nationwide Children's Hospital  Columbus Ohio USA"},{"name":"Department of Pediatrics The Ohio State University  Columbus Ohio USA"},{"name":"Departments of Pathology and Neurology, The Ohio State University  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kelly J.","family":"Lehman","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linda P.","family":"Lowes","sequence":"additional","affiliation":[{"name":"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\/0000-0002-0573-9504","authenticated-orcid":false,"given":"Natalie F.","family":"Reash","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Megan A.","family":"Iammarino","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay N.","family":"Alfano","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah","family":"Lewis","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen","family":"Church","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Shell","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachael A.","family":"Potter","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Danielle A.","family":"Griffin","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Hogan","sequence":"additional","affiliation":[{"name":"Center for Gene Therapy Nationwide Children's Hospital  Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shufang","family":"Wang","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefanie","family":"Mason","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eddie","family":"Darton","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc.  Cambridge Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"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":[[2023,8,14]]},"reference":[{"key":"e_1_2_13_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(09)70271-6"},{"key":"e_1_2_13_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12883-019-1304-8"},{"key":"e_1_2_13_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.05.012"},{"key":"e_1_2_13_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejphar.2023.175675"},{"key":"e_1_2_13_6_1","unstructured":"US Food and Drug Administration.ELEVIDYS\u2122 Highlights of prescribing information.2023https:\/\/www.fda.gov\/media\/169679\/download. Accessed July 2023"},{"key":"e_1_2_13_7_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2020.1484"},{"key":"e_1_2_13_8_1","unstructured":"ClinicalTrials.gov. NCT03375164.A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP\u20109001) in Participants With Duchenne Muscular Dystrophy (DMD).https:\/\/classic.clinicaltrials.gov\/ct2\/show\/NCT03375164. Accessed July 2023"},{"key":"e_1_2_13_9_1","unstructured":"ClinicalTrials.gov. NCT01603407.Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR\u2010DMD).https:\/\/www.clinicaltrials.gov\/ct2\/show\/NCT01603407. Accessed July 2023."},{"key":"e_1_2_13_10_1","unstructured":"FOR\u2010DMD.https:\/\/for-dmd.org\/en. Accessed July 2023."},{"key":"e_1_2_13_11_1","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2022.4315"},{"key":"e_1_2_13_12_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0221097"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27955","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,10]],"date-time":"2024-01-10T23:17:20Z","timestamp":1704928640000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27955"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,8,14]]},"references-count":11,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2024,1]]}},"alternative-id":["10.1002\/mus.27955"],"URL":"https:\/\/doi.org\/10.1002\/mus.27955","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,8,14]]},"assertion":[{"value":"2023-02-11","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-07-22","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-08-14","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}