{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,12]],"date-time":"2026-10-12T20:10:38Z","timestamp":1791835838373,"version":"4.3.4"},"reference-count":31,"publisher":"Wiley","license":[{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>Spinal muscular atrophy (SMA) manifests with progressive motor neuron degeneration, leading to muscle weakness. Onasemnogene abeparvovec is a US Food and Drug Administration\u2010approved gene replacement therapy for SMA. This study aimed to present short\u2010term data of children in the United Arab Emirates (UAE) treated with onasemnogene abeparvovec, particularly in the context of children requiring invasive ventilatory support via tracheostomy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A retrospective analysis was performed on 60 children who received onasemnogene abeparvovec. All these children received corticosteroids. They were followed up for up to 3 months. Motor function assessments were performed before and after the gene therapy. Comprehensive clinical evaluations, including pulmonary functions, were performed at baseline and the 3\u2010month mark.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Forty\u2010three percent were male, and the mean age at the time of infusion was 29.6\u2009months (SD \u00b1\u200917.2). The mean weight was 10.1\u2009kg (SD 2.6). All children demonstrated marked improvements in motor function within 3 months of gene therapy administration. No adverse effects attributable to corticosteroid therapy were observed. Positive clinical outcomes, including increased ventilator\u2010free intervals, reduced antibiotic dependency, and fewer hospital admissions, were reported among children with invasive ventilation via tracheostomy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>This study demonstrates the favorable tolerability and promising responses to onasemnogene abeparvovec in invasively ventilated pediatric patients. Early improvements in motor function, as observed within 3 months post\u2010treatment, suggest its potential as a viable therapeutic option for this vulnerable patient population.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.28222","type":"journal-article","created":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T07:52:35Z","timestamp":1722498755000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Onasemnogene abeparvovec gene therapy for spinal muscular atrophy: A cohort study from the\n                    <scp>United Arab Emirates<\/scp>"],"prefix":"10.1002","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9235-2470","authenticated-orcid":false,"given":"Vivek","family":"Mundada","sequence":"first","affiliation":[{"name":"Department of Paediatric Neuroscience Aster DM Healthcare, Medcare Women and Children Hospital  Dubai UAE"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Omendra","family":"Narayan","sequence":"additional","affiliation":[{"name":"Department of Paediatric Pulmonology American Hospital  Dubai UAE"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Siddharth","family":"Arora","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Neurodisability Aster DM Healthcare, Medcare Women and Children Hospital  Dubai UAE"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nidhi","family":"Beri","sequence":"additional","affiliation":[{"name":"Department of Paediatrics Neurodisability Aster DM Healthcare, Medcare Women and Children Hospital  Dubai UAE"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rania","family":"Abusamra","sequence":"additional","affiliation":[{"name":"Department of Paediatric Pulmonology Mediclinic City Hospital  Dubai UAE"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deepak","family":"Mullasery","sequence":"additional","affiliation":[{"name":"Department of Paediatric Physiotherapy Medcare Physiotherapy and Rehabilitation Centre  Dubai UAE"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deepak","family":"Parashar","sequence":"additional","affiliation":[{"name":"Division of Health Sciences Warwick Medical School, University of Warwick  Coventry UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,8]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1177\/1060028020914274"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-019-01162-5"},{"key":"e_1_2_12_4_1","unstructured":"Novartis Gene Therapies Managed Access Program. Accessed March 8 2024.https:\/\/www.novartis.com\/healthcare-professionals\/managed-accessprograms\/novartis-gene-therapies-managed-access-program"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2021.07.315"},{"key":"e_1_2_12_6_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2020-0729"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2023.07.105"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27363"},{"key":"e_1_2_12_9_1","unstructured":"Summary of Product Characteristics. 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