{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,27]],"date-time":"2026-10-27T12:16:03Z","timestamp":1793103363394,"version":"4.5.0"},"reference-count":30,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,4,20]],"date-time":"2023-04-20T00:00:00Z","timestamp":1681948800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003196","name":"Ministero della Salute","doi-asserted-by":"publisher","award":["GR-2018-12365706"],"award-info":[{"award-number":["GR-2018-12365706"]}],"id":[{"id":"10.13039\/501100003196","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003196","name":"Ministero della Salute","doi-asserted-by":"publisher","award":["RF-2019-12370334"],"award-info":[{"award-number":["RF-2019-12370334"]}],"id":[{"id":"10.13039\/501100003196","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["thelancet.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["eClinicalMedicine"],"published-print":{"date-parts":[[2023,5]]},"DOI":"10.1016\/j.eclinm.2023.101997","type":"journal-article","created":{"date-parts":[[2023,5,5]],"date-time":"2023-05-05T11:38:26Z","timestamp":1683286706000},"page":"101997","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":45,"special_numbering":"C","title":["Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in na\u00efve patients with spinal muscular atrophy and following switch from other therapies"],"prefix":"10.1016","volume":"59","author":[{"given":"Marika","family":"Pane","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beatrice","family":"Berti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4061-4501","authenticated-orcid":false,"given":"Anna","family":"Capasso","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giorgia","family":"Coratti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Varone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adele","family":"D\u2019Amico","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonia","family":"Messina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Riccardo","family":"Masson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valeria Ada","family":"Sansone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Alice","family":"Donati","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Caterina","family":"Agosto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudio","family":"Bruno","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federica","family":"Ricci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonella","family":"Pini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Delio","family":"Gagliardi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Massimiliano","family":"Filosto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stefania","family":"Corti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniela","family":"Leone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Concetta","family":"Palermo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberta","family":"Onesimo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto","family":"De Sanctis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8481-3812","authenticated-orcid":false,"given":"Martina","family":"Ricci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Bitetti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Sframeli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Dosi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emilio","family":"Albamonte","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Ticci","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noemi","family":"Brolatti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Enrico","family":"Bertini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard","family":"Finkel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9851-5365","authenticated-orcid":false,"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Carmela","family":"Pera","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Bravetti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marco","family":"Piastra","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Orazio","family":"Genovese","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gianpaolo","family":"Cicala","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicola","family":"Forcina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Carnicella","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giulia","family":"Stanca","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michele","family":"Sacchini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michela","family":"Catteruccia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michele","family":"Tosi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renato","family":"Cutrera","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudio","family":"Cherchi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Beatrice","family":"Chiarini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Salmin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marina","family":"Pedemonte","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Govoni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irene","family":"Mizzoni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simone","family":"Morando","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Riccardo","family":"Zanin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Enrica","family":"Rolle","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eleonora","family":"Salomon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melania","family":"Giannotta","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gaia","family":"Scarpini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Toscano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eloisa","family":"Gitto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberto","family":"Materia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rossella","family":"D\u2019Alessandro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"5","key":"10.1016\/j.eclinm.2023.101997_bib1","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1016\/S1474-4422(12)70061-3","article-title":"Childhood spinal muscular atrophy: controversies and challenges","volume":"11","author":"Mercuri","year":"2012","journal-title":"Lancet Neurol"},{"issue":"6","key":"10.1016\/j.eclinm.2023.101997_bib2","doi-asserted-by":"crossref","first-page":"883","DOI":"10.1002\/ana.25101","article-title":"Natural history of infantile-onset spinal muscular atrophy","volume":"82","author":"Kolb","year":"2017","journal-title":"Ann Neurol"},{"issue":"9","key":"10.1016\/j.eclinm.2023.101997_bib3","doi-asserted-by":"crossref","first-page":"810","DOI":"10.1212\/WNL.0000000000000741","article-title":"Observational study of spinal muscular atrophy type I and implications for clinical trials","volume":"83","author":"Finkel","year":"2014","journal-title":"Neurology"},{"issue":"1","key":"10.1016\/j.eclinm.2023.101997_bib4","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1038\/s41572-022-00380-8","article-title":"Spinal muscular atrophy","volume":"8","author":"Mercuri","year":"2022","journal-title":"Nat Rev Dis Primers"},{"issue":"18","key":"10.1016\/j.eclinm.2023.101997_bib5","doi-asserted-by":"crossref","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","article-title":"Single-dose gene-replacement therapy for spinal muscular atrophy","volume":"377","author":"Mendell","year":"2017","journal-title":"N\u00a0Engl J Med"},{"issue":"4","key":"10.1016\/j.eclinm.2023.101997_bib6","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1016\/S1474-4422(21)00001-6","article-title":"Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial","volume":"20","author":"Day","year":"2021","journal-title":"Lancet Neurol"},{"issue":"10","key":"10.1016\/j.eclinm.2023.101997_bib7","doi-asserted-by":"crossref","first-page":"832","DOI":"10.1016\/S1474-4422(21)00251-9","article-title":"Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy type 1 (STR1VE-EU): an open-label, single-arm, multicentre, phase 3 trial","volume":"20","author":"Mercuri","year":"2021","journal-title":"Lancet Neurol"},{"issue":"9","key":"10.1016\/j.eclinm.2023.101997_bib8","doi-asserted-by":"crossref","first-page":"1075","DOI":"10.1080\/14712598.2022.2066471","article-title":"Onasemnogene abeparvovec for the treatment of spinal muscular atrophy","volume":"22","author":"McMillan","year":"2022","journal-title":"Expert Opin Biol Ther"},{"issue":"7","key":"10.1016\/j.eclinm.2023.101997_bib9","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1001\/jamaneurol.2021.1272","article-title":"Five-year extension results of the phase 1 START trial of onasemnogene abeparvovec in spinal muscular atrophy","volume":"78","author":"Mendell","year":"2021","journal-title":"JAMA Neurol"},{"issue":"3","key":"10.1016\/j.eclinm.2023.101997_bib10","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1002\/acn3.51519","article-title":"Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy","volume":"9","author":"D'Silva","year":"2022","journal-title":"Ann Clin Transl Neurol"},{"issue":"4","key":"10.1016\/j.eclinm.2023.101997_bib11","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1016\/j.braindev.2021.12.006","article-title":"Short-term clinical outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy","volume":"44","author":"Lee","year":"2022","journal-title":"Brain Dev"},{"issue":"1","key":"10.1016\/j.eclinm.2023.101997_bib12","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/S2352-4642(21)00287-X","article-title":"Gene replacement therapy with onasemnogene abeparvovec in children with spinal muscular atrophy aged 24 months or younger and bodyweight up to 15 kg: an observational cohort study","volume":"6","author":"Weiss","year":"2022","journal-title":"Lancet Child Adolesc Health"},{"issue":"10","key":"10.1016\/j.eclinm.2023.101997_bib13","doi-asserted-by":"crossref","first-page":"794","DOI":"10.1016\/j.nmd.2019.08.014","article-title":"Development of an academic disease registry for spinal muscular atrophy","volume":"29","author":"Mercuri","year":"2019","journal-title":"Neuromuscul Disord"},{"issue":"2","key":"10.1016\/j.eclinm.2023.101997_bib14","first-page":"159","article-title":"Evaluator training and reliability for SMA global nusinersen trials 1","volume":"5","author":"Glanzman","year":"2018","journal-title":"J\u00a0Neuromuscul Dis"},{"issue":"1","key":"10.1016\/j.eclinm.2023.101997_bib15","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1016\/0960-8966(94)00075-K","article-title":"Chaos in the classification of SMA: a possible resolution","volume":"5","author":"Dubowitz","year":"1995","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.eclinm.2023.101997_bib16","series-title":"Nelson textbook of pediatrics","author":"Nelson","year":"2020"},{"issue":"7","key":"10.1016\/j.eclinm.2023.101997_bib17","doi-asserted-by":"crossref","first-page":"1390","DOI":"10.1038\/s41591-022-01867-3","article-title":"Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the phase III SPR1NT trial","volume":"28","author":"Strauss","year":"2022","journal-title":"Nat Med"},{"issue":"7","key":"10.1016\/j.eclinm.2023.101997_bib18","doi-asserted-by":"crossref","first-page":"1381","DOI":"10.1038\/s41591-022-01866-4","article-title":"Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the phase III SPR1NT trial","volume":"28","author":"Strauss","year":"2022","journal-title":"Nat Med"},{"key":"10.1016\/j.eclinm.2023.101997_bib19","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/j.pediatrneurol.2022.05.001","article-title":"Safety of onasemnogene abeparvovec for patients with spinal muscular atrophy 8.5 kg or heavier in a global managed access program","volume":"132","author":"Chand","year":"2022","journal-title":"Pediatr Neurol"},{"issue":"3","key":"10.1016\/j.eclinm.2023.101997_bib20","doi-asserted-by":"crossref","first-page":"560","DOI":"10.1016\/j.jhep.2020.11.001","article-title":"Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy","volume":"74","author":"Chand","year":"2021","journal-title":"J\u00a0Hepatol"},{"issue":"3","key":"10.1016\/j.eclinm.2023.101997_bib21","first-page":"117","article-title":"Switching therapies: safety profile of onasemnogene abeparvovec-xioi in a SMA1 patient previously treated with risdiplam","volume":"41","author":"Tosi","year":"2022","journal-title":"Acta Myol"},{"key":"10.1016\/j.eclinm.2023.101997_bib22","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1089\/hum.2022.161","article-title":"Onasemnogene abeparvovec in type 1 spinal muscular atrophy: a systematic review and meta-analysis","volume":"34","author":"Pascual-Morena","year":"2022","journal-title":"Hum Gene Ther"},{"issue":"2","key":"10.1016\/j.eclinm.2023.101997_bib23","doi-asserted-by":"crossref","first-page":"E8","DOI":"10.1002\/mus.27648","article-title":"Novel use of nusinersen as a therapeutic bridge to onasemnogene abeparvovec-xioi in a premature neonate with type 1 spinal muscular atrophy","volume":"66","author":"Ferrante","year":"2022","journal-title":"Muscle Nerve"},{"issue":"23","key":"10.1016\/j.eclinm.2023.101997_bib24","first-page":"5540","article-title":"Combination therapy with\u00a0nusinersen and onasemnogene abeparvovec-xioi in spinal\u00a0muscular atrophy type I","volume":"10","author":"Mirea","year":"2021","journal-title":"J\u00a0Clin Med"},{"issue":"4","key":"10.1016\/j.eclinm.2023.101997_bib25","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1002\/mus.27375","article-title":"Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1","volume":"64","author":"Oechsel","year":"2021","journal-title":"Muscle Nerve"},{"issue":"4","key":"10.1016\/j.eclinm.2023.101997_bib26","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1002\/mus.27034","article-title":"Combination molecular therapies for type 1 spinal muscular atrophy","volume":"62","author":"Harada","year":"2020","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.eclinm.2023.101997_bib27","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1016\/j.jpeds.2020.11.054","article-title":"Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: a case series","volume":"231","author":"Chand","year":"2021","journal-title":"J\u00a0Pediatr"},{"issue":"19","key":"10.1016\/j.eclinm.2023.101997_bib28","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1212\/WNL.0000000000200676","article-title":"Child neurology: pathologically confirmed thrombotic microangiopathy caused by onasemnogene abeparvovec treatment for SMA","volume":"98","author":"Yazaki","year":"2022","journal-title":"Neurology"},{"key":"10.1016\/j.eclinm.2023.101997_bib29","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.pediatrneurol.2021.01.012","article-title":"Clinical experience with gene therapy in older patients with spinal muscular atrophy","volume":"118","author":"Matesanz","year":"2021","journal-title":"Pediatr Neurol"},{"issue":"10\u201311","key":"10.1016\/j.eclinm.2023.101997_bib30","doi-asserted-by":"crossref","first-page":"676","DOI":"10.1038\/s41434-021-00273-7","article-title":"Gene therapy for spinal muscular atrophy: the Qatari experience","volume":"28","author":"Ali","year":"2021","journal-title":"Gene Ther"}],"container-title":["eClinicalMedicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2589537023001748?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2589537023001748?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,11]],"date-time":"2025-11-11T22:18:13Z","timestamp":1762899493000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2589537023001748"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,5]]},"references-count":30,"alternative-id":["S2589537023001748"],"URL":"https:\/\/doi.org\/10.1016\/j.eclinm.2023.101997","relation":{},"ISSN":["2589-5370"],"issn-type":[{"value":"2589-5370","type":"print"}],"subject":[],"published":{"date-parts":[[2023,5]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in na\u00efve patients with spinal muscular atrophy and following switch from other therapies","name":"articletitle","label":"Article Title"},{"value":"eClinicalMedicine","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.eclinm.2023.101997","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 The Authors. Published by Elsevier Ltd.","name":"copyright","label":"Copyright"}],"article-number":"101997"}}