{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,7]],"date-time":"2026-04-07T20:55:59Z","timestamp":1775595359161,"version":"3.50.1"},"reference-count":15,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2023,7,1]],"date-time":"2023-07-01T00:00:00Z","timestamp":1688169600000},"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"],"published-print":{"date-parts":[[2023,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>New therapeutic strategies to increase survival motor neuron protein levels in spinal muscular atrophy (SMA) have focused on replacing the <jats:italic>SMN1<\/jats:italic> gene. Onasemnogene abeparvovec was approved by the US Food and Drug Administration in 2019 for treatment of children with SMA less than 2\u2009years of age. Postmarketing studies are limited, especially outside of Europe and the United States. Herein we describe a single\u2010center experience with onasemnogene abeparvovec from the Middle East.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Between November 17, 2020 and January 31, 2022, 25 children with SMA received onasemnogene abeparvovec at our center in the United Arab Emirates. Data were collected on patients\u2019 demographics, age at diagnosis, SMA type, genetic information, relevant medical history, laboratory investigations, and Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP\u2010INTEND) functional assessment scores at baseline and 1 and 3 months after gene therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Onasemnogene abeparvovec was well tolerated. Significant improvements in CHOP\u2010INTEND scores were observed after the therapy. Elevation of liver enzymes and thrombocytopenia were the most common adverse events, but were transient and managed with high\u2010dose corticosteroids. There were no life\u2010threatening adverse events or deaths reported during the 3\u2010month follow\u2010up period.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>The study findings concurred with those of previously published studies. Side effects of gene transfer therapy are well tolerated, although serious complications can arise. In such cases, persistent transaminitis for example, steroid dose escalation is warranted with close observation of the patient's clinical status and lab values. Combination therapy should be explored as an alternative to gene transfer therapy only.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27926","type":"journal-article","created":{"date-parts":[[2023,7,1]],"date-time":"2023-07-01T13:30:01Z","timestamp":1688218201000},"page":"269-277","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Gene transfer therapy in children with spinal muscular atrophy: A single\u2010center experience with a cohort of 25 children"],"prefix":"10.1002","volume":"68","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9142-708X","authenticated-orcid":false,"given":"Nidheesh","family":"Chencheri","sequence":"first","affiliation":[{"name":"Neurosciences Center of Excellence Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gail","family":"Alexander","sequence":"additional","affiliation":[{"name":"Neurosciences Center of Excellence Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ahmed","family":"Nugud","sequence":"additional","affiliation":[{"name":"Neurosciences Center of Excellence Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Eurose","family":"Majadas","sequence":"additional","affiliation":[{"name":"Rehabilitation Department Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Haneen","family":"Salim","sequence":"additional","affiliation":[{"name":"Rehabilitation Department Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katy","family":"Prudhomme","sequence":"additional","affiliation":[{"name":"Rehabilitation Department Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noleen","family":"DeJager","sequence":"additional","affiliation":[{"name":"Rehabilitation Department Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vaishnavi Shetty","family":"Janardhanan","sequence":"additional","affiliation":[{"name":"Rehabilitation Department Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Haitham","family":"Elbashir","sequence":"additional","affiliation":[{"name":"Neurosciences Center of Excellence Al Jalila Children's Specialty Hospital  Dubai United Arab Emirates"},{"name":"Pediatric Department Mohammed Bin Rashid University of Medicine and Health Sciences  Dubai United Arab Emirates"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2023,7]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.09.006"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms21093297"},{"key":"e_1_2_12_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.10.002"},{"key":"e_1_2_12_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-017-8549-1"},{"key":"e_1_2_12_6_1","article-title":"250 AVXS\u2010101 phase 3 study in spinal muscular atrophy type 1","volume":"90","author":"Day JW","year":"2019","journal-title":"Journal of Neurology, Neurosurgery & Psychiatry"},{"key":"e_1_2_12_7_1","unstructured":"European Medicines Agency. Zolgensma.2020.https:\/\/www.ema.europa.eu\/en\/medicines\/human\/epar\/zolgensma. Accessed January 5 2022."},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.xcrm.2021.100346"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pedneo.2022.07.006"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.11.014"},{"key":"e_1_2_12_11_1","first-page":"76","article-title":"Adeno\u2010associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec","volume":"21","author":"Day JW","year":"2021","journal-title":"Mol Ther"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2020\u20100729"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-200593"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41434-021-00273-7"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2019.05.005"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27926","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,1,4]],"date-time":"2024-01-04T05:27:19Z","timestamp":1704346039000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27926"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,7]]},"references-count":15,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2023,9]]}},"alternative-id":["10.1002\/mus.27926"],"URL":"https:\/\/doi.org\/10.1002\/mus.27926","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,7]]},"assertion":[{"value":"2022-09-11","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-06-14","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-07-01","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}