{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,24]],"date-time":"2026-10-24T03:31:09Z","timestamp":1792812669378,"version":"4.5.0"},"reference-count":49,"publisher":"Springer Science and Business Media LLC","issue":"9","license":[{"start":{"date-parts":[[2025,6,27]],"date-time":"2025-06-27T00:00:00Z","timestamp":1750982400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2025,6,27]],"date-time":"2025-06-27T00:00:00Z","timestamp":1750982400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"}],"funder":[{"DOI":"10.13039\/100005614","name":"Biogen","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005614","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Adv Ther"],"published-print":{"date-parts":[[2025,9]]},"DOI":"10.1007\/s12325-025-03260-1","type":"journal-article","created":{"date-parts":[[2025,6,27]],"date-time":"2025-06-27T02:41:20Z","timestamp":1750992080000},"page":"4143-4160","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":11,"title":["Systematic Review and Meta-analysis of Long-Term Nusinersen Effectiveness in Adolescents and Adults with Spinal Muscular Atrophy"],"prefix":"10.1007","volume":"42","author":[{"given":"Tim","family":"Hagenacker","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5698-8802","authenticated-orcid":false,"given":"Angela D.","family":"Paradis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katherine A.","family":"Lawson-Michod","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bora","family":"Youn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,6,27]]},"reference":[{"issue":"18","key":"3260_CR1","doi-asserted-by":"publisher","first-page":"1723","DOI":"10.1056\/NEJMoa1702752","volume":"377","author":"RS Finkel","year":"2017","unstructured":"Finkel RS, Mercuri E, Darras BT, et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med. 2017;377(18):1723\u201332.","journal-title":"N Engl J Med"},{"issue":"7","key":"3260_CR2","doi-asserted-by":"publisher","first-page":"625","DOI":"10.1056\/NEJMoa1710504","volume":"378","author":"E Mercuri","year":"2018","unstructured":"Mercuri E, Darras BT, Chiriboga CA, et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378(7):625\u201335.","journal-title":"N Engl J Med"},{"issue":"2","key":"3260_CR3","doi-asserted-by":"publisher","first-page":"157","DOI":"10.1002\/mus.27853","volume":"68","author":"TO Crawford","year":"2023","unstructured":"Crawford TO, Swoboda KJ, De Vivo DC, et al. Continued benefit of nusinersen initiated in the presymptomatic stage of spinal muscular atrophy: 5-year update of the NURTURE study. Muscle Nerve. 2023;68(2):157\u201370.","journal-title":"Muscle Nerve"},{"issue":"5","key":"3260_CR4","doi-asserted-by":"publisher","first-page":"1483","DOI":"10.1007\/s40120-024-00653-2","volume":"13","author":"T Hagenacker","year":"2024","unstructured":"Hagenacker T, Maggi L, Coratti G, et al. Effectiveness of nusinersen in adolescents and adults with spinal muscular atrophy: systematic review and meta-analysis. Neurol Ther. 2024;13(5):1483\u2013504.","journal-title":"Neurol Ther"},{"issue":"1","key":"3260_CR5","doi-asserted-by":"publisher","first-page":"430","DOI":"10.1186\/s13023-021-02065-z","volume":"16","author":"G Coratti","year":"2021","unstructured":"Coratti G, Cutrona C, Pera MC, et al. Motor function in type\u00a02 and 3 SMA patients treated with nusinersen: a critical review and meta-analysis. Orphanet J Rare Dis. 2021;16(1):430.","journal-title":"Orphanet J Rare Dis"},{"issue":"2","key":"3260_CR6","doi-asserted-by":"publisher","first-page":"464","DOI":"10.1007\/s13311-022-01200-3","volume":"19","author":"M Gavriilaki","year":"2022","unstructured":"Gavriilaki M, Moschou M, Papaliagkas V, et al. Nusinersen in adults with 5q spinal muscular atrophy: a systematic review and meta-analysis. Neurotherapeutics. 2022;19(2):464\u201375.","journal-title":"Neurotherapeutics"},{"issue":"5","key":"3260_CR7","doi-asserted-by":"publisher","first-page":"836","DOI":"10.1002\/mus.25120","volume":"54","author":"S Dunaway Young","year":"2016","unstructured":"Dunaway Young S, Montes J, Kramer SS, et al. Six-minute walk test is reliable and valid in spinal muscular atrophy. Muscle Nerve. 2016;54(5):836\u201342.","journal-title":"Muscle Nerve"},{"issue":"4","key":"3260_CR8","doi-asserted-by":"publisher","first-page":"426","DOI":"10.1002\/mus.26419","volume":"59","author":"MC Pera","year":"2019","unstructured":"Pera MC, Coratti G, Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: 12\u00a0month changes. Muscle Nerve. 2019;59(4):426\u201330.","journal-title":"Muscle Nerve"},{"issue":"3-a Suppl","key":"3260_CR9","first-page":"S54","volume":"25","author":"V Williams","year":"2019","unstructured":"Williams V, Stull DE, Houghton K, Williams N, Teynor M. Minimal clinically important differences of the Expanded Hammersmith Functional Motor Scale in later-onset spinal muscular atrophy: results from the phase\u00a03 CHERISH trial. J Manag Care Spec Pharm. 2019;25(3-a Suppl):S54.","journal-title":"J Manag Care Spec Pharm"},{"key":"3260_CR10","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.i4919","volume":"355","author":"JA Sterne","year":"2016","unstructured":"Sterne JA, Hern\u00e1n MA, Reeves BC, et al. ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions. BMJ. 2016;355: i4919.","journal-title":"BMJ"},{"issue":"1","key":"3260_CR11","doi-asserted-by":"publisher","first-page":"62","DOI":"10.1016\/j.braindev.2023.08.005","volume":"46","author":"M Funato","year":"2024","unstructured":"Funato M, Kino A, Iwata R, et al. Later efficacy of nusinersen treatment in adult patients with spinal muscular atrophy: a retrospective case study with a median 4-year follow-up. Brain Dev. 2024;46(1):62\u20137.","journal-title":"Brain Dev"},{"issue":"6","key":"3260_CR12","doi-asserted-by":"publisher","first-page":"2887","DOI":"10.1007\/s10072-024-07515-7","volume":"45","author":"C Serrao","year":"2024","unstructured":"Serrao C, Domingues S, de Campos CF, et al. Nusinersen in adults with type\u00a03 spinal muscular atrophy: long-term outcomes on motor and respiratory function. Neurol Sci. 2024;45(6):2887\u201391.","journal-title":"Neurol Sci"},{"issue":"7","key":"3260_CR13","doi-asserted-by":"publisher","first-page":"2393","DOI":"10.1007\/s10072-023-06698-9","volume":"44","author":"D Arslan","year":"2023","unstructured":"Arslan D, Inan B, Kilinc M, et al. Nusinersen for adults with spinal muscular atrophy. Neurol Sci. 2023;44(7):2393\u2013400.","journal-title":"Neurol Sci"},{"key":"3260_CR14","doi-asserted-by":"publisher","first-page":"1294028","DOI":"10.3389\/fneur.2023.1294028","volume":"14","author":"J Cho","year":"2023","unstructured":"Cho J, Lee J, Kim J, et al. Nusinersen demonstrates effectiveness in treating spinal muscular atrophy: findings from a three-year nationwide study in Korea. Front Neurol. 2023;14:1294028.","journal-title":"Front Neurol"},{"issue":"1","key":"3260_CR15","doi-asserted-by":"publisher","first-page":"119","DOI":"10.1017\/cjn.2024.49","volume":"52","author":"I C\u00f4t\u00e9","year":"2025","unstructured":"C\u00f4t\u00e9 I, Hodgkinson V, Nury M, Bastenier-Boutin L, Rodrigue X. A real-world study of nusinersen effects in adults with spinal muscular atrophy type\u00a02 and 3. Can J Neurol Sci. 2025;52(1):119\u201328.","journal-title":"Can J Neurol Sci"},{"key":"3260_CR16","doi-asserted-by":"publisher","DOI":"10.1016\/j.lanepe.2024.100862","volume":"39","author":"R G\u00fcnther","year":"2024","unstructured":"G\u00fcnther R, Wurster CD, Brakemeier S, et al. Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study. Lancet Reg Health Eur. 2024;39:100862.","journal-title":"Lancet Reg Health Eur"},{"issue":"1","key":"3260_CR17","doi-asserted-by":"publisher","first-page":"230","DOI":"10.1186\/s13023-023-02769-4","volume":"18","author":"A \u0141usakowska","year":"2023","unstructured":"\u0141usakowska A, W\u00f3jcik A, Fr\u0105czek A, et al. Long-term nusinersen treatment across a wide spectrum of spinal muscular atrophy severity: a real-world experience. Orphanet J Rare Dis. 2023;18(1):230.","journal-title":"Orphanet J Rare Dis"},{"issue":"3","key":"3260_CR18","doi-asserted-by":"publisher","first-page":"631","DOI":"10.3390\/neurolint16030047","volume":"16","author":"A AlTawari","year":"2024","unstructured":"AlTawari A, Zakaria M, Kamel W, et al. Nusinersen treatment for spinal muscular atrophy: retrospective multicenter study of pediatric and adult patients in Kuwait. Neurol Int. 2024;16(3):631\u201342.","journal-title":"Neurol Int"},{"issue":"2","key":"3260_CR19","doi-asserted-by":"publisher","first-page":"110","DOI":"10.1016\/j.braindev.2022.10.006","volume":"45","author":"H Iwayama","year":"2023","unstructured":"Iwayama H, Kawahara K, Takagi M, et al. Long-term efficacy of nusinersen and its evaluation in adolescent and adult patients with spinal muscular atrophy types 1 and 2. Brain Dev. 2023;45(2):110\u20136.","journal-title":"Brain Dev"},{"issue":"5","key":"3260_CR20","doi-asserted-by":"publisher","first-page":"399","DOI":"10.1016\/j.nmd.2022.02.001","volume":"32","author":"A Tscherter","year":"2022","unstructured":"Tscherter A, R\u00fcsch CT, Baumann D, et al. Evaluation of real-life outcome data of patients with spinal muscular atrophy treated with nusinersen in Switzerland. Neuromuscul Disord. 2022;32(5):399\u2013409.","journal-title":"Neuromuscul Disord"},{"issue":"11","key":"3260_CR21","doi-asserted-by":"publisher","first-page":"3337","DOI":"10.1111\/ene.15501","volume":"29","author":"JF V\u00e1zquez-Costa","year":"2022","unstructured":"V\u00e1zquez-Costa JF, Povedano M, Nascimiento-Osorio AE, et al. Nusinersen in adult patients with 5q spinal muscular atrophy: a multicenter observational cohorts\u2019 study. Eur J Neurol. 2022;29(11):3337\u201346.","journal-title":"Eur J Neurol"},{"issue":"14","key":"3260_CR22","doi-asserted-by":"publisher","first-page":"e1988","DOI":"10.1212\/WNL.0000000000010540","volume":"95","author":"CA Wijngaarde","year":"2020","unstructured":"Wijngaarde CA, Stam M, Otto LAM, et al. Muscle strength and motor function in adolescents and adults with spinal muscular atrophy. Neurology. 2020;95(14):e1988\u201398.","journal-title":"Neurology"},{"issue":"3","key":"3260_CR23","doi-asserted-by":"publisher","first-page":"512","DOI":"10.1111\/ene.13534","volume":"25","author":"RI Wadman","year":"2018","unstructured":"Wadman RI, Wijngaarde CA, Stam M, et al. Muscle strength and motor function throughout life in a cross-sectional cohort of 180 patients with spinal muscular atrophy types 1c\u20134. Eur J Neurol. 2018;25(3):512\u20138.","journal-title":"Eur J Neurol"},{"issue":"6","key":"3260_CR24","doi-asserted-by":"publisher","first-page":"1109","DOI":"10.1002\/ana.25900","volume":"88","author":"G Coratti","year":"2020","unstructured":"Coratti G, Messina S, Lucibello S, et al. Clinical variability in spinal muscular atrophy type\u00a0III. Ann Neurol. 2020;88(6):1109\u201317.","journal-title":"Ann Neurol"},{"issue":"6","key":"3260_CR25","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0199657","volume":"13","author":"J Montes","year":"2018","unstructured":"Montes J, McDermott MP, Mirek E, et al. Ambulatory function in spinal muscular atrophy: age-related patterns of progression. PLoS ONE. 2018;13(6):e0199657.","journal-title":"PLoS ONE"},{"issue":"2","key":"3260_CR26","doi-asserted-by":"publisher","first-page":"126","DOI":"10.1016\/j.nmd.2015.10.006","volume":"26","author":"E Mercuri","year":"2016","unstructured":"Mercuri E, Finkel R, Montes J, et al. Patterns of disease progression in type\u00a02 and 3 SMA: implications for clinical trials. Neuromuscul Disord. 2016;26(2):126\u201331.","journal-title":"Neuromuscul Disord"},{"issue":"3","key":"3260_CR27","doi-asserted-by":"publisher","first-page":"212","DOI":"10.1016\/j.nmd.2014.11.008","volume":"25","author":"S Sivo","year":"2015","unstructured":"Sivo S, Mazzone E, Antonaci L, et al. Upper limb module in non-ambulant patients with spinal muscular atrophy: 12\u00a0month changes. Neuromuscul Disord. 2015;25(3):212\u20135.","journal-title":"Neuromuscul Disord"},{"issue":"9","key":"3260_CR28","doi-asserted-by":"publisher","first-page":"765","DOI":"10.1016\/j.nmd.2020.07.004","volume":"30","author":"G Coratti","year":"2020","unstructured":"Coratti G, Lucibello S, Pera MC, et al. Gain and loss of abilities in type\u00a0II SMA: a 12-month natural history study. Neuromuscul Disord. 2020;30(9):765\u201371.","journal-title":"Neuromuscul Disord"},{"issue":"12","key":"3260_CR29","first-page":"1253","volume":"93","author":"L Maggi","year":"2022","unstructured":"Maggi L, Bello L, Bonanno S, et al. Adults with spinal muscular atrophy: a large-scale natural history study shows gender effect on disease. J Neurol Neurosurg Psychiatry. 2022;93(12):1253\u201361.","journal-title":"J Neurol Neurosurg Psychiatry"},{"issue":"8","key":"3260_CR30","doi-asserted-by":"publisher","first-page":"624","DOI":"10.1016\/j.nmd.2013.06.001","volume":"23","author":"E Mazzone","year":"2013","unstructured":"Mazzone E, Bianco F, Main M, et al. Six minute walk test in type\u00a0III spinal muscular atrophy: a 12 month longitudinal study. Neuromuscul Disord. 2013;23(8):624\u20138.","journal-title":"Neuromuscul Disord"},{"issue":"9","key":"3260_CR31","doi-asserted-by":"publisher","first-page":"756","DOI":"10.1016\/j.nmd.2020.07.005","volume":"30","author":"G Coratti","year":"2020","unstructured":"Coratti G, Pera MC, Lucibello S, et al. Age and baseline values predict 12 and 24-month functional changes in type\u00a02 SMA. Neuromuscul Disord. 2020;30(9):756\u201364.","journal-title":"Neuromuscul Disord"},{"issue":"8","key":"3260_CR32","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16309","volume":"31","author":"G Coratti","year":"2024","unstructured":"Coratti G, Bovis F, Pera MC, et al. Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: an international study. Eur J Neurol. 2024;31(8):e16309.","journal-title":"Eur J Neurol"},{"issue":"12","key":"3260_CR33","doi-asserted-by":"publisher","first-page":"2586","DOI":"10.1111\/ene.14472","volume":"27","author":"B Stolte","year":"2020","unstructured":"Stolte B, Bois JM, Bolz S, et al. Minimal clinically important differences in functional motor scores in adults with spinal muscular atrophy. Eur J Neurol. 2020;27(12):2586\u201394.","journal-title":"Eur J Neurol"},{"issue":"3","key":"3260_CR34","doi-asserted-by":"publisher","first-page":"404","DOI":"10.1002\/acn3.51514","volume":"9","author":"M Pane","year":"2022","unstructured":"Pane M, Coratti G, Pera MC, et al. Nusinersen efficacy data for 24-month in type\u00a02 and 3 spinal muscular atrophy. Ann Clin Transl Neurol. 2022;9(3):404\u20139.","journal-title":"Ann Clin Transl Neurol"},{"issue":"12","key":"3260_CR35","doi-asserted-by":"publisher","first-page":"3666","DOI":"10.1111\/ene.15542","volume":"29","author":"JF V\u00e1zquez-Costa","year":"2022","unstructured":"V\u00e1zquez-Costa JF, Povedano M, Nascimiento-Osorio AE, et al. Validation of motor and functional scales for the evaluation of adult patients with 5q spinal muscular atrophy. Eur J Neurol. 2022;29(12):3666\u201375.","journal-title":"Eur J Neurol"},{"issue":"6","key":"3260_CR36","doi-asserted-by":"publisher","first-page":"1223","DOI":"10.1002\/mus.28269","volume":"70","author":"B Stolte","year":"2024","unstructured":"Stolte B, Neuhoff S, Lipka J, et al. Performance fatigability in adults with spinal muscular atrophy treated long-term with nusinersen. Muscle Nerve. 2024;70(6):1223\u20139.","journal-title":"Muscle Nerve"},{"issue":"5","key":"3260_CR37","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000213371","volume":"104","author":"I Cordts","year":"2025","unstructured":"Cordts I, Fuetterer C, Wachinger A, et al. Long-term dynamics of CSF and serum neurofilament light chain in adult patients with 5q spinal muscular atrophy treated with nusinersen. Neurology. 2025;104(5):e213371.","journal-title":"Neurology"},{"key":"3260_CR38","unstructured":"Grigoli EG, Zanin R, Masson R, Maggi L, Bonanno S. A seven-year real-world experience with nusinersen treatment in adult SMA patients at C. Besta Neurological Institute (poster presented at 54\u00b0 Congresso Societ\u00e0 Italiana di Neurologia). 2024."},{"issue":"3","key":"3260_CR39","doi-asserted-by":"publisher","first-page":"244","DOI":"10.3390\/jpm14030244","volume":"14","author":"A Belancic","year":"2024","unstructured":"Belancic A, Strbad T, Kucan Stiglic M, Vitezic D. Switching from nusinersen to risdiplam: a Croatian real-world experience on effectiveness and safety. J Pers Med. 2024;14(3):244.","journal-title":"J Pers Med"},{"issue":"1","key":"3260_CR40","doi-asserted-by":"publisher","first-page":"95","DOI":"10.1111\/bcp.16267","volume":"91","author":"A Belancic","year":"2025","unstructured":"Belancic A, Faour AK, Gkrinia EMM, Vitezic D. A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy. Br J Clin Pharmacol. 2025;91(1):95\u2013116.","journal-title":"Br J Clin Pharmacol"},{"issue":"4","key":"3260_CR41","doi-asserted-by":"publisher","first-page":"317","DOI":"10.1016\/S1474-4422(20)30037-5","volume":"19","author":"T Hagenacker","year":"2020","unstructured":"Hagenacker T, Wurster CD, G\u00fcnther R, et al. Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study. Lancet Neurol. 2020;19(4):317\u201325.","journal-title":"Lancet Neurol"},{"issue":"4","key":"3260_CR42","doi-asserted-by":"publisher","first-page":"453","DOI":"10.3233\/JND-190416","volume":"6","author":"MC Walter","year":"2019","unstructured":"Walter MC, Wenninger S, Thiele S, et al. Safety and treatment effects of nusinersen in longstanding adult 5q-SMA type\u00a03\u00a0\u2013 a prospective observational study. J Neuromuscul Dis. 2019;6(4):453\u201365.","journal-title":"J Neuromuscul Dis"},{"issue":"9","key":"3260_CR43","doi-asserted-by":"publisher","first-page":"6004","DOI":"10.1007\/s00415-024-12567-y","volume":"271","author":"N Wang","year":"2024","unstructured":"Wang N, Hu Y, Jiao K, et al. Long-term impact of nusinersen on motor and electrophysiological outcomes in adolescent and adult spinal muscular atrophy: insights from a multicenter retrospective study. J Neurol. 2024;271(9):6004\u201314.","journal-title":"J Neurol"},{"issue":"7","key":"3260_CR44","doi-asserted-by":"publisher","first-page":"3810","DOI":"10.3390\/ijms25073810","volume":"25","author":"P Andr\u00e9s-Benito","year":"2024","unstructured":"Andr\u00e9s-Benito P, V\u00e1zquez-Costa JF, \u00d1ungo Garz\u00f3n NC, et al. Neurodegeneration biomarkers in adult spinal muscular atrophy (SMA) patients treated with nusinersen. Int J Mol Sci. 2024;25(7):3810.","journal-title":"Int J Mol Sci"},{"key":"3260_CR45","doi-asserted-by":"publisher","first-page":"28","DOI":"10.1016\/j.nmd.2024.01.005","volume":"36","author":"N Simsek Erdem","year":"2024","unstructured":"Simsek Erdem N, Gunes Gencer GY, Alaamel A, Uysal H. Effect of nusinersen treatment on quality of life and motor function in adult patients with spinal muscular atrophy. Neuromuscul Disord. 2024;36:28\u201332.","journal-title":"Neuromuscul Disord"},{"key":"3260_CR46","doi-asserted-by":"publisher","first-page":"42","DOI":"10.1016\/j.nmd.2024.05.003","volume":"41","author":"G Coratti","year":"2024","unstructured":"Coratti G, Civitello M, Rohwer A, et al. Changes in abilities over the initial 12\u00a0months of nusinersen treatment for type\u00a0II SMA. Neuromuscul Disord. 2024;41:42\u201350.","journal-title":"Neuromuscul Disord"},{"issue":"8","key":"3260_CR47","doi-asserted-by":"publisher","first-page":"241","DOI":"10.1007\/s00018-023-04885-7","volume":"80","author":"I Faravelli","year":"2023","unstructured":"Faravelli I, Gagliardi D, Abati E, et al. Multi-omics profiling of CSF from spinal muscular atrophy type\u00a03 patients after nusinersen treatment: a 2-year follow-up multicenter retrospective study. Cell Mol Life Sci. 2023;80(8):241.","journal-title":"Cell Mol Life Sci"},{"issue":"8","key":"3260_CR48","doi-asserted-by":"publisher","first-page":"2839","DOI":"10.3390\/jcm12082839","volume":"12","author":"A Belan\u010di\u0107","year":"2023","unstructured":"Belan\u010di\u0107 A, Strbad T, Ku\u010dan \u0160tigli\u0107 M, Vitezi\u0107 D. Effectiveness of nusinersen in type\u00a01, 2 and 3 spinal muscular atrophy: Croatian real-world data. J Clin Med. 2023;12(8):2839.","journal-title":"J Clin Med"},{"issue":"11","key":"3260_CR49","doi-asserted-by":"publisher","first-page":"1166","DOI":"10.1136\/jnnp-2020-323822","volume":"91","author":"L Maggi","year":"2020","unstructured":"Maggi L, Bello L, Bonanno S, et al. Nusinersen safety and effects on motor function in adult spinal muscular atrophy type\u00a02 and 3. J Neurol Neurosurg Psychiatry. 2020;91(11):1166\u201374.","journal-title":"J Neurol Neurosurg Psychiatry"}],"container-title":["Advances in Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-025-03260-1.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s12325-025-03260-1\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-025-03260-1.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,9,7]],"date-time":"2025-09-07T00:57:09Z","timestamp":1757206629000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s12325-025-03260-1"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,6,27]]},"references-count":49,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2025,9]]}},"alternative-id":["3260"],"URL":"https:\/\/doi.org\/10.1007\/s12325-025-03260-1","relation":{},"ISSN":["0741-238X","1865-8652"],"issn-type":[{"value":"0741-238X","type":"print"},{"value":"1865-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,6,27]]},"assertion":[{"value":"14 March 2025","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"22 May 2025","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"27 June 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Tim Hagenacker received grants\/research support from Biogen and Roche, and honoraria or consulting fees from Biogen and Roche. Angela D. Paradis and Bora Youn are employees of Biogen and may hold stock in the company. Katherine A. Lawson-Michod reports personal fees from Epidemiologic Research & Methods, LLC.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"This article is based on previously conducted studies and does not contain any new studies with human participants or animals performed by any of the authors. Informed consent was not required for this study.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}