{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,18]],"date-time":"2026-09-18T20:40:14Z","timestamp":1789764014955,"version":"4.0.1"},"reference-count":41,"publisher":"SAGE Publications","issue":"23-24","license":[{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2024,12,1]],"date-time":"2024-12-01T00:00:00Z","timestamp":1733011200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2024,12]]},"abstract":"<jats:p>\n                    Delandistrogene moxeparvovec is a gene transfer therapy for Duchenne muscular dystrophy (DMD) that uses an adeno-associated viral vector to deliver a micro-dystrophin transgene to skeletal and cardiac muscle. This study evaluated the long-term survival and cardiac efficacy of delandistrogene moxeparvovec in a DMD-mutated (DMD\n                    <jats:sup>MDX<\/jats:sup>\n                    ) rat model of DMD-related cardiomyopathy. DMD\n                    <jats:sup>MDX<\/jats:sup>\n                    male rats, aged 21\u201342 days, were injected with 1.33 \u00d7 10\n                    <jats:sup>14<\/jats:sup>\n                    viral genomes\/kilogram (vg\/kg) delandistrogene moxeparvovec and followed for 12, 24, and 52 weeks. Ambulation was recorded\n                    <jats:italic toggle=\"yes\">via<\/jats:italic>\n                    the Photobeam Activity System, whereas echocardiograms, cardiomyocyte contractility, calcium handling, and histological analysis of fibrosis were used to evaluate cardiac disease at 12-, 24-, and 52-weeks post-treatment. A separate cohort of rats was used to assess the impact of delandistrogene moxeparvovec on survival. Treatment with delandistrogene moxeparvovec extended median survival in DMD\n                    <jats:sup>MDX<\/jats:sup>\n                    rats to &gt;25 months versus the 13-month median survival in saline-control-treated DMD\n                    <jats:sup>MDX<\/jats:sup>\n                    rats. Compared with saline control, delandistrogene moxeparvovec therapy elicited statistically significant improvements across cardiac parameters approaching wild-type values with additional benefits in mobility, histopathology, and fibrosis observed. Transgene expression was maintained up to &gt;25 months and micro-dystrophin expression was broadly distributed across skeletal and cardiac muscle. Taken together, these findings demonstrate long-term cardiac efficacy and improved survival following delandistrogene moxeparvovec treatment in DMD\n                    <jats:sup>MDX<\/jats:sup>\n                    rats.\n                  <\/jats:p>","DOI":"10.1089\/hum.2024.013","type":"journal-article","created":{"date-parts":[[2024,11,28]],"date-time":"2024-11-28T12:06:42Z","timestamp":1732795602000},"page":"978-988","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":13,"title":["Long-Term Survival and Myocardial Function Following Systemic Delivery of Delandistrogene Moxeparvovec in DMD\n                    <sup>MDX<\/sup>\n                    Rats"],"prefix":"10.1177","volume":"35","author":[{"given":"Stephen","family":"Baine","sequence":"first","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"Wier","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luke","family":"Lemmerman","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Grace","family":"Cooper-Olson","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amber","family":"Kempton","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alex","family":"Haile","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julian","family":"Endres","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Fedoce","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ellyn","family":"Nesbit","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Louise R.","family":"Rodino-Klapac","sequence":"additional","affiliation":[{"name":"Sarepta Therapeutics, Inc., Cambridge, Massachusetts, 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"}]}],"member":"179","published-online":{"date-parts":[[2024,12,13]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.3233\/JND-210678"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-021-00248-3"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamacardio.2016.4910"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1212\/wnl.0000000000012910"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0631-3"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacbts.2021.11.004"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.3389\/fcvm.2022.952755"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.3390\/jcm9020520"},{"key":"e_1_3_5_10_2","unstructured":"US Food & Drug Administration. ELEVIDYS Highlights of Prescribing Information 2023. Available from: https:\/\/www.fda.gov\/media\/169679\/download [Last accessed: September 2023]."},{"key":"e_1_3_5_11_2","unstructured":"Sarepta Therapeutics. Sarepta Therapeutics Announces FDA Approval of ELEVIDYS the First Gene Therapy to Treat Duchenne Muscular Dystrophy 2023. Available from: https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-announces-fda-approval-elevidys-first-gene [Last accessed: July 2023]."},{"key":"e_1_3_5_12_2","unstructured":"UAE Ministry of Health & Prevention. Registered Medical Product Directory Dubai. UAE Ministry of Health and Prevention; 2023. Available from: https:\/\/mohap.gov.ae\/en\/services\/registered-medical-product-directory [Last accessed: February 2024]."},{"key":"e_1_3_5_13_2","unstructured":"NHRA Bahrain. Pharmacy & Pharmaceutical Products Regulation (PPR) 2024. 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