{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,25]],"date-time":"2026-09-25T23:28:42Z","timestamp":1790378922649,"version":"4.1.0"},"reference-count":25,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2022,2,16]],"date-time":"2022-02-16T00:00:00Z","timestamp":1644969600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000925","name":"National Health and Medical Research Council","doi-asserted-by":"publisher","award":["APP1194940"],"award-info":[{"award-number":["APP1194940"]}],"id":[{"id":"10.13039\/501100000925","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2022,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real\u2010world practice, in a broad population of infants with spinal muscular atrophy (SMA).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A prospective cohort study of children with SMA treated with onasemnogene abeparvovec at Sydney Children's Hospital Network, Australia was conducted from August 2019 to November 2021. Safety outcomes included clinical and laboratory evaluations. Efficacy assessments included World Health Organisation (WHO) motor milestones, oral and swallowing abilities, and requirements for respiratory support. The implementation of a model of care for onasemnogene abeparvovec administration in health practice is described.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>21 children were treated (age range, 0.65\u201324\u2009months; body weight range, 2.5\u201312.5\u2009kg) and 19\/21 (90.4%) had previous nusinersen. Transient treatment\u2010related side effects occurred in all children; vomiting (100%), transaminitis (57%) and thrombocytopaenia (33%). Incidence of moderate\/severe transaminitis was significantly greater in infants weighing \u22658\u2009kg compared with &lt;8\u2009kg (<jats:italic>p<\/jats:italic>\u2009&lt;\u20090.05). Duration of prednisolone following treatment was prolonged (mean 87.5\u2009days, range 57\u2013274\u2009days). 16\/21 (76%) children gained at least one WHO motor milestone. Stabilisation or improvement in bulbar or respiratory function was observed in 20\/21 (95.2%) patients. Implementation challenges were mitigated by developing standard operating procedures and facilitating exchange of knowledge.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>This study provides real\u2010world evidence to inform treatment decisions and guide therapeutic expectations for onasemnogene abeparvovec and combination therapy for SMA in health practice, especially for children weighing \u22658\u2009kg receiving higher vector loads. Proactive clinical and laboratory surveillance is essential to facilitate individualised management of risks.<\/jats:p><\/jats:sec>","DOI":"10.1002\/acn3.51519","type":"journal-article","created":{"date-parts":[[2022,2,16]],"date-time":"2022-02-16T09:03:06Z","timestamp":1645002186000},"page":"339-350","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":73,"title":["Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy"],"prefix":"10.1002","volume":"9","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6265-3053","authenticated-orcid":false,"given":"Arlene M.","family":"D'Silva","sequence":"first","affiliation":[{"name":"Department of Neurology Sydney Children's Hospital Network Sydney New South Wales Australia"},{"name":"School of Clinical Medicine, UNSW Medicine &amp; Health, Randwick Clinical Campus, Discipline of Paediatrics University of New South Wales Sydney New South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra","family":"Holland","sequence":"additional","affiliation":[{"name":"Department of Neurology Sydney Children's Hospital Network Sydney New South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4176-4010","authenticated-orcid":false,"given":"Didu","family":"Kariyawasam","sequence":"additional","affiliation":[{"name":"Department of Neurology Sydney Children's Hospital Network Sydney New South Wales Australia"},{"name":"School of Clinical Medicine, UNSW Medicine &amp; Health, Randwick Clinical Campus, Discipline of Paediatrics University of New South Wales Sydney New South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karen","family":"Herbert","sequence":"additional","affiliation":[{"name":"Physiotherapy Sydney Children's Hospital Sydney New South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Barclay","sequence":"additional","affiliation":[{"name":"Department of Pharmacy Prince of Wales Hospital, Sydney Children's Hospital Randwick and The Royal Hospital for Women Randwick New\u00a0South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anita","family":"Cairns","sequence":"additional","affiliation":[{"name":"Neurosciences Department Queensland's Children Hospital South Brisbane Queensland Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suzanna C.","family":"MacLennan","sequence":"additional","affiliation":[{"name":"Neurology Department Women's &amp; Children's Hospital North Adelaide South Australia Australia"},{"name":"School of Paediatrics and Reproductive Health University of Adelaide Adelaide South Australia Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monique M.","family":"Ryan","sequence":"additional","affiliation":[{"name":"Department of Neurology Royal Children's Hospital Melbourne Australia"},{"name":"Neuroscience Research Group Murdoch Children's Research Institute Melbourne Australia"},{"name":"Department of Paediatrics The University of Melbourne Melbourne Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hugo","family":"Sampaio","sequence":"additional","affiliation":[{"name":"Department of Neurology Sydney Children's Hospital Network Sydney New South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicholas","family":"Smith","sequence":"additional","affiliation":[{"name":"Gene Therapy Research Unit Sydney Children's Hospitals Network and Children's Medical Research Institute, The University of Sydney Westmead Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ian R.","family":"Woodcock","sequence":"additional","affiliation":[{"name":"Department of Neurology Royal Children's Hospital Melbourne Australia"},{"name":"Neuroscience Research Group Murdoch Children's Research Institute Melbourne Australia"},{"name":"Department of Paediatrics The University of Melbourne Melbourne Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eppie M.","family":"Yiu","sequence":"additional","affiliation":[{"name":"Department of Neurology Royal Children's Hospital Melbourne Australia"},{"name":"Neuroscience Research Group Murdoch Children's Research Institute Melbourne Australia"},{"name":"Department of Paediatrics The University of Melbourne Melbourne Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ian E.","family":"Alexander","sequence":"additional","affiliation":[{"name":"Gene Therapy Research Unit Sydney Children's Hospitals Network and Children's Medical Research Institute, The University of Sydney Westmead Australia"},{"name":"Discipline of Child and Adolescent Health, Sydney Medical School, Faculty of Medicine and Health The University of Sydney Sydney New\u00a0South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle A.","family":"Farrar","sequence":"additional","affiliation":[{"name":"Department of Neurology Sydney Children's Hospital Network Sydney New South Wales Australia"},{"name":"School of Clinical Medicine, UNSW Medicine &amp; 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