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The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. 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(2022). The Safety and Efficacy of Nusinersen in the Treatment of Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Medicina, 58.","DOI":"10.3390\/medicina58020213"},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"668","DOI":"10.1002\/mus.27187","article-title":"Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study","volume":"63","author":"Acsadi","year":"2021","journal-title":"Muscle Nerve"},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","article-title":"Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy","volume":"377","author":"Finkel","year":"2017","journal-title":"N. Engl. J. Med."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"625","DOI":"10.1056\/NEJMoa1710504","article-title":"Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy","volume":"378","author":"Mercuri","year":"2018","journal-title":"N. Engl. J. Med."},{"key":"ref_5","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":"ref_6","doi-asserted-by":"crossref","first-page":"915","DOI":"10.1056\/NEJMoa2009965","article-title":"Risdiplam in type 1 spinal muscular atrophy","volume":"384","author":"Baranello","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"347","DOI":"10.2217\/cer-2021-0216","article-title":"How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: A systematic literature review and indirect treatment comparison","volume":"11","author":"Ribero","year":"2022","journal-title":"J. Comp. Eff. Res."},{"key":"ref_8","unstructured":"UK NICE Guidelines Committee (2022, May 14). Nusinersen for treating spinal muscular atrophy [Internet]. United Kingdom\u2019s National Institute for Health and Care Excellence; 2019 Jul. Report No.: TA588., Available online: https:\/\/www.nice.org.uk\/guidance\/ta588."}],"container-title":["Medicina"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/1648-9144\/58\/6\/766\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,10]],"date-time":"2025-10-10T23:24:52Z","timestamp":1760138692000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/1648-9144\/58\/6\/766"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,6,6]]},"references-count":8,"journal-issue":{"issue":"6","published-online":{"date-parts":[[2022,6]]}},"alternative-id":["medicina58060766"],"URL":"https:\/\/doi.org\/10.3390\/medicina58060766","relation":{},"ISSN":["1648-9144"],"issn-type":[{"type":"electronic","value":"1648-9144"}],"subject":[],"published":{"date-parts":[[2022,6,6]]}}}