{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,20]],"date-time":"2026-03-20T10:29:03Z","timestamp":1774002543738,"version":"3.50.1"},"reference-count":11,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2025,11,27]],"date-time":"2025-11-27T00:00:00Z","timestamp":1764201600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,11,27]],"date-time":"2025-11-27T00:00:00Z","timestamp":1764201600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Gene Ther"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>Duchenne Muscular Dystrophy is a rare, X-linked neuromuscular disorder that leads to progressive muscle degeneration, loss of ambulation, and premature mortality due to respiratory and cardiac failure. Historically, Duchennke Muscular Dystrophy has been managed through supportive and symptomatic treatments, with limited options for disease modification. However, advancements in gene therapy have introduced promising interventions aimed at addressing the underlying dystrophin deficiency. Delandistrogene moxeparvovec (Elevidys) received accelerated approval from the U.S. Food and Drug Administration in June 2023 for ambulatory children aged 4\u20135 years with a confirmed diagnosis of Duchenne Muscular Dystrophy. This approval represented an advancement, offering a disease-modifying therapy at an early stage when muscle function remains relatively preserved. The Food and Drug Administration expanded its approval in June 2024 to include both ambulatory and non-ambulatory children aged 4 years and older. This study provides a retrospective real-world analysis of eight Duchenne Muscular Dystrophy patients who received Elevidys gene therapy at our center in Qatar. Recognizing the complexities involved in treating older Duchenne Muscular Dystrophy patients, a standardized protocol for pre- and post-infusion care was implemented. Our findings highlight the positive clinical outcomes of gene therapy for Duchenne Muscular Dystrophy patients in Qatar.<\/jats:p>","DOI":"10.1038\/s41434-025-00580-3","type":"journal-article","created":{"date-parts":[[2025,11,27]],"date-time":"2025-11-27T16:44:31Z","timestamp":1764261871000},"page":"78-83","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Real-world experience with gene therapy in Duchenne muscular dystrophy center readiness and patients safety: report from Qatar"],"prefix":"10.1038","volume":"33","author":[{"given":"Mahmoud Fawzi","family":"Osman","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Khalid","family":"Ibrahim","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Claire","family":"Gleeson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Haytham","family":"Ibrahim","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ikram Ul","family":"Haque","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noora","family":"Alhamad","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8522-7738","authenticated-orcid":false,"given":"Tawfeg","family":"Ben-Omran","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2025,11,27]]},"reference":[{"key":"580_CR1","doi-asserted-by":"publisher","first-page":"2025","DOI":"10.1016\/S0140-6736(19)32910-1","volume":"394","author":"E Mercuri","year":"2019","unstructured":"Mercuri E, B\u00f6nnemann CG, Muntoni F. Muscular dystrophies. Lancet. 2019;394:2025\u201338. https:\/\/doi.org\/10.1016\/S0140-6736(19)32910-1.","journal-title":"Lancet"},{"key":"580_CR2","doi-asserted-by":"publisher","first-page":"482","DOI":"10.1016\/j.nmd.2014.03.008","volume":"24","author":"JK Mah","year":"2014","unstructured":"Mah JK, Korngut L, Dykeman J, Day L, Pringsheim T, Jette N. A systematic review and meta-analysis on the epidemiology of Duchenne and Becker muscular dystrophy. Neuromuscul Disord. 2014;24:482\u201391. https:\/\/doi.org\/10.1016\/j.nmd.2014.03.008.","journal-title":"Neuromuscul Disord"},{"key":"580_CR3","doi-asserted-by":"publisher","first-page":"743","DOI":"10.1016\/j.neubiorev.2013.03.016","volume":"37","author":"WM Snow","year":"2013","unstructured":"Snow WM, Anderson JE, Jakobson LS. Neuropsychological and neurobehavioral functioning in Duchenne muscular dystrophy: A review. Neurosci Biobehav Rev. 2013;37:743\u201352.","journal-title":"Neurosci Biobehav Rev"},{"key":"580_CR4","doi-asserted-by":"publisher","first-page":"145","DOI":"10.1136\/jmedgenet-2015-103387","volume":"53","author":"A Aartsma-Rus","year":"2016","unstructured":"Aartsma-Rus A, Ginjaar IB, Bushby K. The importance of genetic diagnosis for Duchenne muscular dystrophy. J Med Genet. 2016;53:145\u201351.","journal-title":"J Med Genet"},{"key":"580_CR5","doi-asserted-by":"publisher","first-page":"3647","DOI":"10.1093\/hmg\/ddw210","volume":"25","author":"J Zhao","year":"2016","unstructured":"Zhao J, Kodippili K, Yue Y, Hakim CH, Wasala L, Pan X, et al. Dystrophin contains multiple independent membrane-binding domains. Hum Mol Genet. 2016;25:3647\u201353. https:\/\/doi.org\/10.1093\/hmg\/ddw210.","journal-title":"Hum Mol Genet"},{"key":"580_CR6","doi-asserted-by":"publisher","first-page":"251","DOI":"10.1016\/S1474-4422(18)30024-3","volume":"17","author":"DJ Birnkrant","year":"2018","unstructured":"Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17:251\u201367. https:\/\/doi.org\/10.1016\/S1474-4422(18)30024-3.","journal-title":"Lancet Neurol"},{"key":"580_CR7","doi-asserted-by":"publisher","first-page":"11","DOI":"10.1016\/j.pediatrneurol.2024.01.003","volume":"153","author":"JR Mendell","year":"2024","unstructured":"Mendell JR, Proud C, Zaidman CM, Mason S, Darton E, Wang S, et al. Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy. Pediatr Neurol. 2024;153:11\u20138. https:\/\/doi.org\/10.1016\/j.pediatrneurol.2024.01.003.","journal-title":"Pediatr Neurol"},{"key":"580_CR8","doi-asserted-by":"publisher","first-page":"1122","DOI":"10.1001\/jamaneurol.2020.1484","volume":"77","author":"JR Mendell","year":"2020","unstructured":"Mendell JR, Sahenk Z, Lehman K, Nease C, Lowes LP, Miller NF, et al. Assessment of systemic delivery of rAAVrh74.MHCK7.micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial. JAMA Neurol. 2020;77:1122\u201331. https:\/\/doi.org\/10.1001\/jamaneurol.2020.1484.","journal-title":"JAMA Neurol"},{"key":"580_CR9","doi-asserted-by":"publisher","first-page":"332","DOI":"10.1038\/s41591-024-03304-z","volume":"31","author":"JR Mendell","year":"2025","unstructured":"Mendell JR, Muntoni F, McDonald CM, Mercuri EM, Ciafaloni E, Komaki H, et al. AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial. Nat Med. 2025;31:332\u201341. https:\/\/doi.org\/10.1038\/s41591-024-03304-z.","journal-title":"Nat Med"},{"key":"580_CR10","doi-asserted-by":"publisher","first-page":"464","DOI":"10.1016\/j.ymthe.202012.007","volume":"29","author":"JR Mendell","year":"2021","unstructured":"Mendell JR, Al-Zaidy S, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, et al. Current clinical applications of in Vivo Gene Therapy with AAVs. Mol Ther. 2021;29:464\u201388. https:\/\/doi.org\/10.1016\/j.ymthe.202012.007.","journal-title":"Mol Ther"},{"key":"580_CR11","unstructured":"Sarepta Therapeutics Inc. Elevidys (delandistrogene moxeparvovec) [prescribing information]. Cambridge, MA: Sarepta Therapeutics Inc; 2024."}],"container-title":["Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/s41434-025-00580-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41434-025-00580-3","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/s41434-025-00580-3.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,2,25]],"date-time":"2026-02-25T00:04:41Z","timestamp":1771977881000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/s41434-025-00580-3"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,11,27]]},"references-count":11,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["580"],"URL":"https:\/\/doi.org\/10.1038\/s41434-025-00580-3","relation":{},"ISSN":["0969-7128","1476-5462"],"issn-type":[{"value":"0969-7128","type":"print"},{"value":"1476-5462","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,11,27]]},"assertion":[{"value":"13 July 2025","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"17 October 2025","order":2,"name":"revised","label":"Revised","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"7 November 2025","order":3,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"27 November 2025","order":4,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"The authors declare no competing interests.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}},{"value":"Legal guardians of all patients signed an informed consent after a detailed discussion of the potential benefits, risks, and alternative treatment options for gene therapy. This consent process ensured that families were fully aware of the nature of the therapy, the pre- and post-infusion requirements, and the importance of regular follow-up. All study methods performed in this study was based on approved treatment protocols and guidelines utilized in Qatar and worldwide in accordance with the Helsinki Declaration. (Sidra ethics board ID: SDR600204 and ethical approval number: 1997345).","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}}]}}