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This systematic literature review aimed to synthesize existing evidence on therapy switching in SMA, focusing on clinical effectiveness and practical implications to support evidence\u2010informed decision\u2010making in a rapidly evolving therapeutic landscape. The review followed PRISMA guidelines and was registered with PROSPERO (CRD42024600221). A systematic search of PubMed\/MEDLINE, Global Health and Embase was conducted between 11 and 14 October 2024. Eligible studies included clinical trials and real\u2010world evidence (RWE) reports describing patients with genetically confirmed SMA who received nusinersen or risdiplam and subsequently switched to nusinersen, risdiplam or onasemnogene abeparvovec. Four studies met the inclusion criteria\u2014three RWE studies and one clinical trial. The variability in measures of central tendency and variability among studies precluded the calculation of pooled summary values. Nonetheless, switching treatments was generally associated with stable motor function, with some improvements reported in selected outcome measures; ventilatory and nutritional support requirements remained largely unchanged. However, long\u2010term outcomes and standardized data were limited. Future research should prioritize robust RWE and post\u2010marketing surveillance to evaluate long\u2010term safety and effectiveness, incorporate standardized switching protocols, and account for SMA genotype\u2013phenotype variation.<\/jats:p>","DOI":"10.1002\/bcp.70145","type":"journal-article","created":{"date-parts":[[2025,6,20]],"date-time":"2025-06-20T06:24:59Z","timestamp":1750400699000},"source":"Crossref","is-referenced-by-count":6,"title":["Switching disease\u2010modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes"],"prefix":"10.1002","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7848-6600","authenticated-orcid":false,"given":"Andrej","family":"Belan\u010di\u0107","sequence":"first","affiliation":[{"name":"Department of Basic and Clinical Pharmacology with Toxicology University of Rijeka, Faculty of Medicine  Rijeka Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-1255-8653","authenticated-orcid":false,"given":"Elvira Meni Maria","family":"Gkrinia","sequence":"additional","affiliation":[{"name":"Independent researcher  Athens Greece"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8522-1122","authenticated-orcid":false,"given":"Patrick","family":"Eustaquio","sequence":"additional","affiliation":[{"name":"Independent researcher  Manila Philippines"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0006-9039-0197","authenticated-orcid":false,"given":"Andrea Katrin","family":"Faour","sequence":"additional","affiliation":[{"name":"Independent researcher  Vancouver British Columbia Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6295-6580","authenticated-orcid":false,"given":"Dinko","family":"Vitezi\u0107","sequence":"additional","affiliation":[{"name":"Department of Basic and Clinical Pharmacology with Toxicology University of Rijeka, Faculty of Medicine  Rijeka Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,6,19]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2015.03.010"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010017\u20100671\u20108"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms21093297"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2017.52"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.05.011"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40271\u2010020\u201000415\u2010w"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1710504"},{"key":"e_1_2_9_10_1","unstructured":"Spinraza \u2010 European Medicines Agency.European Medicines Agency. 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