{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,18]],"date-time":"2026-07-18T11:12:07Z","timestamp":1784373127815,"version":"3.55.0"},"reference-count":13,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2019,12,13]],"date-time":"2019-12-13T00:00:00Z","timestamp":1576195200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2020,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>Clinical trials data concerning use of nusinersen in older spinal muscular atrophy (SMA) patients is lacking. We describe our center's experience in using intrathecal nusinersen for older patients in the clinical setting.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Retrospective study.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Twelve patients (12\u201352\u2009years old) were treated with nusinersen. Mean follow\u2010up duration was 17.4\u2009months (range, 4\u201326\u2009months). All patients had scoliosis; 10 had spinal fusion\/instrumentation. All procedures (30 cervical and 57 lumbar punctures) were technically successful. The only side effects were postprocedural headache (9%) and site pain (5.7%). Functional assessments showed stability in 6\/9 patients and improvement in 3\/9 patients. Subjective improvements in endurance, hand strength, and bulbar functioning critical for activities of daily living were reported in 8\/12 patients. None of the patients has discontinued treatment so far.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>Intrathecal nusinersen can be safely delivered in older SMA patients. Available functional outcome measures are not adequate to capture meaningful subjective improvements.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.26769","type":"journal-article","created":{"date-parts":[[2019,11,27]],"date-time":"2019-11-27T01:59:15Z","timestamp":1574819955000},"page":"222-226","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":61,"title":["Nusinersen for older patients with spinal muscular atrophy: A real\u2010world clinical setting experience"],"prefix":"10.1002","volume":"61","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3065-3956","authenticated-orcid":false,"given":"Aravindhan","family":"Veerapandiyan","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Division of Neurology Arkansas Children's Hospital, University of Arkansas for Medical Sciences  Little Rock Arkansas"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katy","family":"Eichinger","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine University of Rochester Medical Center  Rochester New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Debra","family":"Guntrum","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine University of Rochester Medical Center  Rochester New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Kwon","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Pediatric Neurology University of Wisconsin School of Medicine and Public Health  Madison Wisconsin"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay","family":"Baker","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine University of Rochester Medical Center  Rochester New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erin","family":"Collins","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine University of Rochester Medical Center  Rochester New York"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma","family":"Ciafaloni","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine University of Rochester Medical Center  Rochester New York"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2019,12,13]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1710504"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000007527"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25430"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.880131309"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.25120"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000006502"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1177\/1756286418803246"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-018-9124-0"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000006006"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2018.05.010"},{"key":"e_1_2_8_13_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e318271f7e4"},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26419"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.26769","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.26769","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.26769","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,8]],"date-time":"2023-09-08T06:47:46Z","timestamp":1694155666000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.26769"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,12,13]]},"references-count":13,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2020,2]]}},"alternative-id":["10.1002\/mus.26769"],"URL":"https:\/\/doi.org\/10.1002\/mus.26769","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,12,13]]},"assertion":[{"value":"2019-08-26","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-11-23","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2019-12-13","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}