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We searched PubMed, EMBASE, Web of Science and Cochrane through April 2022. Ten reports enrolling 250 SMA patients were included. CHOP INTEND and motor\u2010milestone significant improvements were detected at both short\u2010 and long\u2010term follow\u2010up. Common adverse events included pyrexia, vomiting, thrombocytopenia and elevated aminotransferases. Thrombocytopenia (79.3%, 95%<jats:italic>CI<\/jats:italic>: 65.8~90.5) and elevated aminotransferases (71.7%, 95%<jats:italic>CI<\/jats:italic>: 62.5~80.1) were more common in SMA patients aged older than 8 months. Despite the paucity of randomized control trial data and low quality of evidence to establish the safety and efficacy of onasemnogene abeparvovec in the treatment of SMA, the data suggest that it is a valuable option for patients with this condition.<\/jats:p>","DOI":"10.1111\/jpc.16340","type":"journal-article","created":{"date-parts":[[2023,2,1]],"date-time":"2023-02-01T12:12:47Z","timestamp":1675253567000},"page":"431-438","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":28,"title":["Safety and efficacy of gene therapy with onasemnogene abeparvovec in the treatment of spinal muscular atrophy: A systematic review and meta\u2010analysis"],"prefix":"10.1111","volume":"59","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-8107-1625","authenticated-orcid":false,"given":"Dongling","family":"Yang","sequence":"first","affiliation":[{"name":"Ruikang Clinical Medical College Guangxi University of Chinese Medicine  Nanning Guangxi China"},{"name":"Department of Pediatric Neurology Maternal and Child Health Hospital of Guangxi Zhuang Autonomous Region, Guangxi Clinical Research Center for Pediatric Diseases  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0857-196X","authenticated-orcid":false,"given":"Yiyan","family":"Ruan","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology Maternal and Child Health Hospital of Guangxi Zhuang Autonomous Region, Guangxi Clinical Research Center for Pediatric Diseases  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuyi","family":"Chen","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology Maternal and Child Health Hospital of Guangxi Zhuang Autonomous Region, Guangxi Clinical Research Center for Pediatric Diseases  Nanning Guangxi China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,2]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60921-6"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0724-z"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.005"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-3-319-89689-2_2"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(12)70061-3"},{"key":"e_1_2_9_7_1","unstructured":"AveXis Inc.ZOLGENSMA\u00ae (onasemnogene abeparvovec\u2010xioi): US Prescribing Information; 2019. 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