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This review aims to systematically map the global pipeline of investigational treatments for SMA. Using\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/ClinicalTrials.gov\">ClinicalTrials.gov<\/jats:ext-link>\n                    and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting novel agents, alternative dosing and delivery routes. Inclusion criteria focused on unapproved therapies or new uses of existing drugs, with rigorous data extraction across demographics, modalities and trial phases. Results reveal a shift towards dual\u2010pronged strategies: refining SMN\u2010targeted interventions and expanding SMN\u2010independent approaches, such as myostatin inhibitors, neuromuscular modulators and ion\u2010channel blockers. Early\u2010phase outcomes suggest promising motor function improvements and acceptable safety profiles, though long\u2010term efficacy remains under investigation. This evolving landscape underscores the importance of registry\u2010based analyses in tracking translational innovation, informing stakeholders and guiding future research priorities.\n                  <\/jats:p>","DOI":"10.1002\/bcp.70502","type":"journal-article","created":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T22:16:32Z","timestamp":1773008192000},"page":"1552-1566","source":"Crossref","is-referenced-by-count":1,"title":["The evolving therapeutic landscape of spinal muscular atrophy \u2013 A scoping review of investigational agents, emerging delivery technologies and strategic innovations"],"prefix":"10.1002","volume":"92","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 and Toxicology, Faculty of Medicine University of Rijeka  Rijeka 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