{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T20:10:36Z","timestamp":1792440636902,"version":"4.5.0"},"reference-count":39,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T00:00:00Z","timestamp":1777593600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T00:00:00Z","timestamp":1777593600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,10,27]],"date-time":"2025-10-27T00:00:00Z","timestamp":1761523200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100005614","name":"Biogen Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005614","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":[[2026,5]]},"DOI":"10.1016\/j.nmd.2025.106256","type":"journal-article","created":{"date-parts":[[2025,10,25]],"date-time":"2025-10-25T01:30:39Z","timestamp":1761355839000},"page":"106256","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"special_numbering":"C","title":["A prospective, multi-center, observational study of the safety, tolerability and effectiveness of Nusinersen in adult patients with spinal muscular atrophy"],"prefix":"10.1016","volume":"62","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1255-5877","authenticated-orcid":false,"given":"Craig M.","family":"Zaidman","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Crystal","family":"Proud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bing","family":"Liao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nassim","family":"Rad","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Doreen","family":"Ho","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary-Lynn","family":"Chu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shafeeq","family":"Ladha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas O.","family":"Crawford","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shakti","family":"Nayar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela","family":"Genge","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Margaret","family":"Frey","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chad","family":"Heatwole","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daphne","family":"Lew","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.nmd.2025.106256_bib0001","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":"10.1016\/j.nmd.2025.106256_bib0002","unstructured":"Biogen. SPINRAZA (nusinersen) injection, for intrathecal use: US prescribing information. 2016 [cited 2025 02\/24\/2025]."},{"key":"10.1016\/j.nmd.2025.106256_bib0003","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":"10.1016\/j.nmd.2025.106256_bib0004","doi-asserted-by":"crossref","first-page":"842","DOI":"10.1016\/j.nmd.2019.09.007","article-title":"Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: interim efficacy and safety results from the phase 2 NURTURE study","volume":"29","author":"De Vivo","year":"2019","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2025.106256_bib0005","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1007\/s13311-022-01200-3","article-title":"Nusinersen in adults with 5q spinal muscular atrophy: a systematic review and meta-analysis","volume":"19","author":"Gavriilaki","year":"2022","journal-title":"Neurotherapeutics"},{"issue":"iii","key":"10.1016\/j.nmd.2025.106256_bib0006","article-title":"Aids to the investigation of peripheral nerve injuries. Medical research council: nerve Injuries Research Committee","volume":"48","author":"Compston","year":"1942","journal-title":"His Majesty's Stationery Office"},{"key":"10.1016\/j.nmd.2025.106256_bib0007","doi-asserted-by":"crossref","first-page":"869","DOI":"10.1002\/mus.25430","article-title":"Revised upper limb module for spinal muscular atrophy: development of a new module","volume":"55","author":"Mazzone","year":"2017","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.nmd.2025.106256_bib0008","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0172346","article-title":"Revised Hammersmith Scale for spinal muscular atrophy: a SMA specific clinical outcome assessment tool","volume":"12","author":"Ramsey","year":"2017","journal-title":"PLoS One"},{"key":"10.1016\/j.nmd.2025.106256_bib0009","doi-asserted-by":"crossref","first-page":"111","DOI":"10.1164\/ajrccm.166.1.at1102","article-title":"ATS statement: guidelines for the six-minute walk test","volume":"166","year":"2002","journal-title":"Am J Respir Crit Care Med"},{"key":"10.1016\/j.nmd.2025.106256_bib0010","doi-asserted-by":"crossref","first-page":"836","DOI":"10.1002\/mus.25120","article-title":"Six-minute walk test is reliable and valid in spinal muscular atrophy","volume":"54","author":"Dunaway Young","year":"2016","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.nmd.2025.106256_bib0011","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1183\/09031936.05.00034805","article-title":"Standardisation of spirometry","volume":"26","author":"Miller","year":"2005","journal-title":"Eur Respir J"},{"key":"10.1016\/j.nmd.2025.106256_bib0012","first-page":"725","article-title":"Changes in the normal maximal expiratory flow-volume curve with growth and aging","volume":"127","author":"Knudson","year":"1983","journal-title":"Am Rev Respir Dis"},{"key":"10.1016\/j.nmd.2025.106256_bib0013","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1002\/mus.27223","article-title":"The Spinal Muscular Atrophy Health Index: a novel outcome for measuring how a patient feels and functions","volume":"63","author":"Zizzi","year":"2021","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.nmd.2025.106256_bib0014","doi-asserted-by":"crossref","first-page":"276","DOI":"10.1002\/mus.27644","article-title":"Assessing perspectives of disease burden and clinically meaningful changes using the Spinal Muscular Atrophy Health Index in adolescents and young adults","volume":"66","author":"Mazzella","year":"2022","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.nmd.2025.106256_bib0015","doi-asserted-by":"crossref","first-page":"425","DOI":"10.3233\/JND-160168","article-title":"What matters most: a perspective from adult spinal muscular atrophy patients","volume":"3","author":"Hunter","year":"2016","journal-title":"J Neuromuscul Dis"},{"key":"10.1016\/j.nmd.2025.106256_bib0016","doi-asserted-by":"crossref","first-page":"e1206","DOI":"10.1212\/WNL.0000000000006241","article-title":"Patient reported impact of symptoms in spinal muscular atrophy (PRISM-SMA)","volume":"91","author":"Mongiovi","year":"2018","journal-title":"Neurology"},{"key":"10.1016\/j.nmd.2025.106256_bib0017","first-page":"25","article-title":"Neurodegeneration biomarkers in adult spinal muscular atrophy (SMA) patients treated with Nusinersen","author":"Andres-Benito","year":"2024","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.nmd.2025.106256_bib0018","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1017\/cjn.2024.49","article-title":"A real-world study of nusinersen effects in adults with spinal muscular atrophy type 2 and 3","volume":"52","author":"Cote","year":"2025","journal-title":"Can J Neurol Sci"},{"key":"10.1016\/j.nmd.2025.106256_bib0019","article-title":"Long-term efficacy and safety of nusinersen in adults with 5q spinal muscular atrophy: a prospective European multinational observational study","volume":"39","author":"Gunther","year":"2024","journal-title":"Lancet Reg Health Eur"},{"key":"10.1016\/j.nmd.2025.106256_bib0020","doi-asserted-by":"crossref","first-page":"404","DOI":"10.1002\/acn3.51514","article-title":"Nusinersen efficacy data for 24-month in type 2 and 3 spinal muscular atrophy","volume":"9","author":"Pane","year":"2022","journal-title":"Ann Clin Transl Neurol"},{"key":"10.1016\/j.nmd.2025.106256_bib0021","doi-asserted-by":"crossref","first-page":"631","DOI":"10.3390\/neurolint16030047","article-title":"Nusinersen treatment for spinal muscular atrophy: retrospective multicenter study of pediatric and adult patients in Kuwait","volume":"16","author":"AlTawari","year":"2024","journal-title":"Neurol Int"},{"key":"10.1016\/j.nmd.2025.106256_bib0022","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":"Lusakowska","year":"2023","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.nmd.2025.106256_bib0023","doi-asserted-by":"crossref","first-page":"29","DOI":"10.3233\/JND-221600","article-title":"Improvements in walking distance during Nusinersen treatment - A prospective 3-year SMArtCARE Registry study","volume":"10","author":"Pechmann","year":"2023","journal-title":"J Neuromuscul Dis"},{"key":"10.1016\/j.nmd.2025.106256_bib0024","doi-asserted-by":"crossref","DOI":"10.1016\/j.nmd.2024.08.006","article-title":"Upper limb function changes over 12 months in untreated SMA II and III individuals: an item-level analysis using the Revised Upper Limb Module","volume":"44","author":"Coratti","year":"2024","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2025.106256_bib0025","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1016\/j.nmd.2021.10.009","article-title":"Revised upper limb module in type II and III spinal muscular atrophy: 24-month changes","volume":"32","author":"Coratti","year":"2022","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2025.106256_bib0026","doi-asserted-by":"crossref","first-page":"426","DOI":"10.1002\/mus.26419","article-title":"Revised upper limb module for spinal muscular atrophy: 12 month changes","volume":"59","author":"Pera","year":"2019","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.nmd.2025.106256_bib0027","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0199657","article-title":"Ambulatory function in spinal muscular atrophy: age-related patterns of progression","volume":"13","author":"Montes","year":"2018","journal-title":"PLoS One"},{"key":"10.1016\/j.nmd.2025.106256_bib0028","doi-asserted-by":"crossref","DOI":"10.3390\/jcm12051920","article-title":"2-Year change in revised Hammersmith scale scores in a large cohort of untreated paediatric type 2 and 3 SMA participants","volume":"12","author":"Stimpson","year":"2023","journal-title":"J Clin Med"},{"key":"10.1016\/j.nmd.2025.106256_bib0029","doi-asserted-by":"crossref","first-page":"644","DOI":"10.1212\/01.wnl.0000324623.89105.c4","article-title":"Natural history of SMA IIIb: muscle strength decreases in a predictable sequence and magnitude","volume":"71","author":"Deymeer","year":"2008","journal-title":"Neurology"},{"key":"10.1016\/j.nmd.2025.106256_bib0030","doi-asserted-by":"crossref","first-page":"1069","DOI":"10.1016\/j.nmd.2012.06.352","article-title":"Change in muscle strength over time in spinal muscular atrophy types II and III. A long-term follow-up study","volume":"22","author":"Werlauff","year":"2012","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2025.106256_bib0031","doi-asserted-by":"crossref","first-page":"1889","DOI":"10.1212\/WNL.0b013e318271f7e4","article-title":"Prospective cohort study of spinal muscular atrophy types 2 and 3","volume":"79","author":"Kaufmann","year":"2012","journal-title":"Neurology"},{"key":"10.1016\/j.nmd.2025.106256_bib0032","doi-asserted-by":"crossref","first-page":"359","DOI":"10.1002\/acn3.51281","article-title":"Natural history of type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study","volume":"8","author":"Annoussamy","year":"2021","journal-title":"Ann Clin Transl Neurol"},{"key":"10.1016\/j.nmd.2025.106256_bib0033","first-page":"1181","article-title":"[Spinal muscular atrophy. A 4-year prospective, multicenter, longitudinal study (168 cases)]","volume":"189","author":"Barois","year":"2005","journal-title":"Bull Acad Natl Med"},{"key":"10.1016\/j.nmd.2025.106256_bib0034","doi-asserted-by":"crossref","DOI":"10.3389\/fneur.2023.1294028","article-title":"Nusinersen demonstrates effectiveness in treating spinal muscular atrophy: findings from a three-year nationwide study in Korea","volume":"14","author":"Cho","year":"2023","journal-title":"Front Neurol"},{"key":"10.1016\/j.nmd.2025.106256_bib0035","doi-asserted-by":"crossref","first-page":"2398","DOI":"10.1007\/s00415-020-09847-8","article-title":"Treatment expectations and patient-reported outcomes of nusinersen therapy in adult spinal muscular atrophy","volume":"267","author":"Osmanovic","year":"2020","journal-title":"J Neurol"},{"key":"10.1016\/j.nmd.2025.106256_bib0036","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1007\/s40263-019-00656-w","article-title":"An integrated safety analysis of infants and children with symptomatic spinal muscular atrophy (SMA) treated with Nusinersen in seven clinical trials","volume":"33","author":"Darras","year":"2019","journal-title":"CNS Drugs"},{"key":"10.1016\/j.nmd.2025.106256_bib0037","doi-asserted-by":"crossref","first-page":"902","DOI":"10.1002\/mus.27217","article-title":"Laboratory monitoring of nusinersen safety","volume":"63","author":"Goedeker","year":"2021","journal-title":"Muscle Nerve"},{"key":"10.1016\/j.nmd.2025.106256_bib0038","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1016\/j.nmd.2022.02.001","article-title":"Evaluation of real-life outcome data of patients with spinal muscular atrophy treated with nusinersen in Switzerland","volume":"32","author":"Tscherter","year":"2022","journal-title":"Neuromuscul Disord"},{"key":"10.1016\/j.nmd.2025.106256_bib0039","series-title":"Exploring higher doses of nusinersen in spinal muscular atrophy: final results from parts B and C of the 3-part","author":"Crawford","year":"2024"}],"container-title":["Neuromuscular Disorders"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960896625009836?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0960896625009836?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,4,28]],"date-time":"2026-04-28T02:43:36Z","timestamp":1777344216000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0960896625009836"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,5]]},"references-count":39,"alternative-id":["S0960896625009836"],"URL":"https:\/\/doi.org\/10.1016\/j.nmd.2025.106256","relation":{},"ISSN":["0960-8966"],"issn-type":[{"value":"0960-8966","type":"print"}],"subject":[],"published":{"date-parts":[[2026,5]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"A prospective, multi-center, observational study of the safety, tolerability and effectiveness of Nusinersen in adult patients with spinal muscular atrophy","name":"articletitle","label":"Article Title"},{"value":"Neuromuscular Disorders","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.nmd.2025.106256","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":"106256"}}