{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T01:40:25Z","timestamp":1790991625927,"version":"4.1.0"},"reference-count":31,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2025,10,1]],"date-time":"2025-10-01T00:00:00Z","timestamp":1759276800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,10,1]],"date-time":"2025-10-01T00:00:00Z","timestamp":1759276800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,8,6]],"date-time":"2025-08-06T00:00:00Z","timestamp":1754438400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004336","name":"Novartis AG","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004336","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","nmd-journal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Neuromuscular Disorders"],"published-print":{"date-parts":[[2025,10]]},"DOI":"10.1016\/j.nmd.2025.105454","type":"journal-article","created":{"date-parts":[[2025,8,5]],"date-time":"2025-08-05T21:14:37Z","timestamp":1754428477000},"page":"105454","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"special_numbering":"C","title":["Real-world experience with switch to onasemnogene abeparvovec after initial therapy with nusinersen or risdiplam"],"prefix":"10.1016","volume":"55","author":[{"given":"Magdalena","family":"Chro\u015bci\u0144ska-Krawczyk","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilona","family":"Kozio\u0142","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ewa","family":"Zienkiewicz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.nmd.2025.105454_bib0001","doi-asserted-by":"crossref","first-page":"2155","DOI":"10.1002\/acn3.51889","article-title":"Combination disease-modifying treatment in spinal muscular atrophy: a proposed classification","volume":"10","author":"Proud","year":"2023","journal-title":"Ann Clin Transl Neurol"},{"key":"10.1016\/j.nmd.2025.105454_bib0002","article-title":"Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom","volume":"37","author":"Gowda","year":"2024","journal-title":"Lancet Reg Health Eur"},{"key":"10.1016\/j.nmd.2025.105454_bib0003","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":"Wei\u00df","year":"2022","journal-title":"Lancet Child Adolesc Health"},{"key":"10.1016\/j.nmd.2025.105454_bib0004","doi-asserted-by":"crossref","DOI":"10.1016\/j.eclinm.2023.101997","article-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","volume":"59","author":"Pane","year":"2023","journal-title":"EClinicalMedicine"},{"key":"10.1016\/j.nmd.2025.105454_bib0005","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1016\/j.ejpn.2024.06.001","article-title":"2024 update: European consensus statement on gene therapy for spinal muscular atrophy","volume":"51","author":"Kirschner","year":"2024","journal-title":"Eur J Paediatr Neurol"},{"key":"10.1016\/j.nmd.2025.105454_bib0006","article-title":"Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study","volume":"47","author":"Wei\u00df","year":"2024","journal-title":"Lancet Reg Health Eur"},{"key":"10.1016\/j.nmd.2025.105454_bib0007","doi-asserted-by":"crossref","first-page":"2419","DOI":"10.1212\/WNL.0000000000202541","article-title":"Real-world outcomes of nusinersen or onasemnogene abeparvovec (OA) monotherapy, or switching to OA from nusinersen in SMA patients aged \u22656 months (P7-9.001)","volume":"100","author":"Dabbous","year":"2023","journal-title":"Neurology"},{"key":"10.1016\/j.nmd.2025.105454_bib0008","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1186\/s12883-023-03133-6","article-title":"Treatment of spinal muscular atrophy with onasemnogene abeparvovec in Switzerland: a prospective observational case series study","volume":"23","author":"Stettner","year":"2023","journal-title":"BMC Neurol"},{"key":"10.1016\/j.nmd.2025.105454_bib0009","doi-asserted-by":"crossref","first-page":"5540","DOI":"10.3390\/jcm10235540","article-title":"Combination therapy with nusinersen and onasemnogene abeparvovec-xioi in spinal muscular atrophy type I","volume":"10","author":"Mirea","year":"2021","journal-title":"J Clin Med"},{"key":"10.1016\/j.nmd.2025.105454_bib0010","doi-asserted-by":"crossref","first-page":"155","DOI":"10.1016\/j.nmd.2009.11.014","article-title":"The children's hospital of Philadelphia infant test of neuromuscular disorders (CHOP INTEND): test development and reliability","volume":"20","author":"Glanzman","year":"2010","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2025.105454_bib0011","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1016\/j.nmd.2007.05.009","article-title":"An expanded version of the Hammersmith functional motor scale for SMA II and III patients","volume":"17","author":"O'Hagen","year":"2007","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2025.105454_bib0012","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 Engl J Med"},{"key":"10.1016\/j.nmd.2025.105454_bib0013","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"},{"key":"10.1016\/j.nmd.2025.105454_bib0014","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1056\/NEJMoa2102047","article-title":"Risdiplam-treated infants with type 1 spinal muscular atrophy versus historical controls","volume":"385","author":"Darras","year":"2021","journal-title":"N Engl J Med"},{"key":"10.1016\/j.nmd.2025.105454_bib0015","doi-asserted-by":"crossref","first-page":"425","DOI":"10.3233\/JND-230122","article-title":"Real-world outcomes in patients with spinal muscular atrophy treated with onasemnogene abeparvovec monotherapy: findings from the RESTORE registry","volume":"11","author":"Servais","year":"2024","journal-title":"J Neuromuscul Dis"},{"key":"10.1016\/j.nmd.2025.105454_bib0016","doi-asserted-by":"crossref","DOI":"10.1542\/peds.2020-0729","article-title":"Gene therapy for spinal muscular atrophy: safety and early outcomes","volume":"146","author":"Waldrop","year":"2020","journal-title":"Pediatrics"},{"key":"10.1016\/j.nmd.2025.105454_bib0017","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1016\/j.pediatrneurol.2019.05.005","article-title":"Impact of age and motor function in a phase 1\/2A study of infants with SMA type 1 receiving single-dose gene replacement therapy","volume":"98","author":"Lowes","year":"2019","journal-title":"Pediatr Neurol"},{"key":"10.1016\/j.nmd.2025.105454_bib0018","unstructured":"New novartis phase III data demonstrate meaningful efficacy and safety results of intrathecal onasemnogene abeparvovec in broad patient population with SMA [internet]. Basel: Novartis; 2025 [Available from: https:\/\/www.novartis.com\/news\/media-releases\/new-novartis-phase-iii-data-demonstrate-meaningful-efficacy-and-safety-results-intrathecal-onasemnogene-abeparvovec-broad-patient-population-sma."},{"key":"10.1016\/j.nmd.2025.105454_bib0019","article-title":"Gene-based therapy for the treatment of spinal muscular atrophy types 1 and 2: a systematic review and meta-analysis","author":"Chongmelaxme","year":"2024","journal-title":"Gene Ther"},{"key":"10.1016\/j.nmd.2025.105454_bib0020","doi-asserted-by":"crossref","DOI":"10.1016\/j.clineuro.2025.108983","article-title":"Real-world data on the effect of long-term treatment with nusinersen over > 4 years in a cohort of Swiss patients with spinal muscular atrophy","volume":"255","author":"Tscherter","year":"2025","journal-title":"Clin Neurol Neurosurg"},{"key":"10.1016\/j.nmd.2025.105454_bib0021","doi-asserted-by":"crossref","first-page":"230","DOI":"10.1186\/s13023-023-02769-4","article-title":"Long-term nusinersen treatment across a wide spectrum of spinal muscular atrophy severity: a real-world experience","volume":"18","author":"\u0141usakowska","year":"2023","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.nmd.2025.105454_bib0022","doi-asserted-by":"crossref","DOI":"10.1111\/ene.70268","article-title":"Longitudinal assessment of 4-Year HFMSE changes in SMA II and III patients treated with nusinersen","volume":"32","author":"Coratti","year":"2025","journal-title":"Eur J Neurol"},{"key":"10.1016\/j.nmd.2025.105454_bib0023","doi-asserted-by":"crossref","DOI":"10.3389\/fneur.2025.1541507","article-title":"Nusinersen for children with type I spinal muscular atrophy: 4 years' clinical experience in Turkish cohort","volume":"16","author":"Bekta\u015f","year":"2025","journal-title":"Front Neurol"},{"key":"10.1016\/j.nmd.2025.105454_bib0024","doi-asserted-by":"crossref","first-page":"548","DOI":"10.1002\/acn3.51276","article-title":"Type I SMA \"new natural history\": long-term data in nusinersen-treated patients","volume":"8","author":"Pane","year":"2021","journal-title":"Ann Clin Transl Neurol"},{"key":"10.1016\/j.nmd.2025.105454_bib0025","doi-asserted-by":"crossref","first-page":"fcac269","DOI":"10.1093\/braincomms\/fcac269","article-title":"Population-based assessment of nusinersen efficacy in children with spinal muscular atrophy: a 3-year follow-up study","volume":"4","author":"Scheijmans","year":"2022","journal-title":"Brain Commun"},{"key":"10.1016\/j.nmd.2025.105454_bib0026","first-page":"1","article-title":"Onasemnogene abeparvovec therapy following nusinersen treatment in eight patients with spinal muscular atrophy type 1","volume":"16","author":"Mikos","year":"2021","journal-title":"Open Access J Neurol Neurosurg"},{"key":"10.1016\/j.nmd.2025.105454_bib0027","article-title":"Gene therapy for spinal muscular atrophy: timing is key","volume":"47","author":"Servais","year":"2024","journal-title":"Lancet Reg Health Eur"},{"key":"10.1016\/j.nmd.2025.105454_bib0028","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1016\/j.nmd.2024.07.004","article-title":"Risk-benefit profile of onasemnogene abeparvovec in older and heavier children with spinal muscular atrophy type 1","volume":"42","author":"Finnegan","year":"2024","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2025.105454_bib0029","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1212\/WNL.0000000000200676","article-title":"Pathologically confirmed thrombotic microangiopathy caused by onasemnogene abeparvovec treatment for SMA","volume":"98","author":"Yazaki","year":"2022","journal-title":"Neurology"},{"key":"10.1016\/j.nmd.2025.105454_bib0030","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 Pediatr"},{"key":"10.1016\/j.nmd.2025.105454_bib0031","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0309666","article-title":"Treatment preferences in spinal muscular atrophy: a swing weighting study for caregivers of patients with SMA types 1 and 2","volume":"19","author":"Patel","year":"2024","journal-title":"PLoS One"}],"container-title":["Neuromuscular Disorders"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960896625001816?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960896625001816?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,3,7]],"date-time":"2026-03-07T10:00:16Z","timestamp":1772877616000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0960896625001816"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,10]]},"references-count":31,"alternative-id":["S0960896625001816"],"URL":"https:\/\/doi.org\/10.1016\/j.nmd.2025.105454","relation":{},"ISSN":["0960-8966"],"issn-type":[{"value":"0960-8966","type":"print"}],"subject":[],"published":{"date-parts":[[2025,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Real-world experience with switch to onasemnogene abeparvovec after initial therapy with nusinersen or risdiplam","name":"articletitle","label":"Article Title"},{"value":"Neuromuscular Disorders","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.nmd.2025.105454","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier B.V.","name":"copyright","label":"Copyright"}],"article-number":"105454"}}