{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,31]],"date-time":"2026-10-31T02:35:52Z","timestamp":1793414152019,"version":"4.5.0"},"reference-count":33,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2022,7,24]],"date-time":"2022-07-24T00:00:00Z","timestamp":1658620800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100003359","name":"Generalitat Valenciana","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003359","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004587","name":"Instituto de Salud Carlos III","doi-asserted-by":"publisher","award":["JR19\/00030"],"award-info":[{"award-number":["JR19\/00030"]}],"id":[{"id":"10.13039\/501100004587","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004587","name":"Instituto de Salud Carlos III","doi-asserted-by":"publisher","award":["PI21\/00737"],"award-info":[{"award-number":["PI21\/00737"]}],"id":[{"id":"10.13039\/501100004587","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004587","name":"Instituto de Salud Carlos III","doi-asserted-by":"publisher","award":["PI19\/01178"],"award-info":[{"award-number":["PI19\/01178"]}],"id":[{"id":"10.13039\/501100004587","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Euro J of Neurology"],"published-print":{"date-parts":[[2022,11]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background and purpose<\/jats:title>\n                    <jats:p>The aim was to assess the safety and efficacy of nusinersen in adult 5q spinal muscular atrophy (SMA) patients.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Patients older than 15\u2009years and followed for at least 6\u00a0months with one motor scale (Hammersmith Functional Motor Scale Expanded, HFMSE; Revised Upper Limb Module, RULM) in five referral centers were included. The clinical and patients' global impression of change (CGI\u2010C and PGI\u2010C) were recorded in treated patients at the last visit. Functional scales (Egen Klassification, EK2; Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, ALSFRS\u2010R) and the percentage predicted forced vital capacity were collected when available.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Seventy\u2010nine SMA patients (39 treated with nusinersen) were included. Compared with untreated patients, treated patients showed a significant improvement of 2 points (\u00b10.46) in RULM (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.001) after 6 months. After a mean follow\u2010up of 16\u2009months, nusinersen treatment was associated with a significant improvement in HFMSE (odds ratio [OR]\u00a01.15,\n                      <jats:italic>p<\/jats:italic>\n                      \u00a0=\u00a00.006), the 6\u2010min walk test (OR\u00a0=\u00a01.07,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.001) and the EK2 (OR\u00a0=\u00a00.81,\n                      <jats:italic>p<\/jats:italic>\n                      \u00a0=\u00a00.001). Compared with untreated patients, more treated patients experienced clinically meaningful improvements in all scales, but these differences were statistically significant only for RULM (\n                      <jats:italic>p<\/jats:italic>\n                      \u00a0=\u00a00.033), ALSFRS\u2010R (\n                      <jats:italic>p<\/jats:italic>\n                      \u00a0=\u00a00.005) and EK2 (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009&lt;\u20090.001). According to the CGI\u2010C and PGI\u2010C, 64.1% and 61.5% of treated patients improved with treatment. Being a non\u2010sitter was associated with less response to treatment, whilst a longer time of treatment was associated with better response. Most treated patients (77%) presented at least one adverse event, mostly mild.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Nusinersen treatment is associated with some improvements in adult SMA patients. Most severely affected patients with complex spines are probably those with the most unfavorable risk\u2013benefit ratio.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ene.15501","type":"journal-article","created":{"date-parts":[[2022,7,25]],"date-time":"2022-07-25T01:34:32Z","timestamp":1658712872000},"page":"3337-3346","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":36,"title":["Nusinersen in adult patients with 5q spinal muscular atrophy: A\u00a0multicenter observational cohorts' study"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3043-7938","authenticated-orcid":false,"given":"Juan F.","family":"V\u00e1zquez\u2010Costa","sequence":"first","affiliation":[{"name":"Neuromuscular Unit, Department of Neurology Hospital Universitario y Polit\u00e9cnico la Fe  Valencia Spain"},{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Raras (CIBERER)  Valencia Spain"},{"name":"Department of Medicine Universitat de Val\u00e8ncia  Valencia Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M\u00f3nica","family":"Povedano","sequence":"additional","affiliation":[{"name":"Motor Neuron Unit, Neurology Department Bellvitge Hospital\u2010IDIBELL  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andr\u00e9s E.","family":"Nascimiento\u2010Osorio","sequence":"additional","affiliation":[{"name":"Neuromuscular Unit, Neuropediatric Department Institut de Recerca Pedi\u00e0trica Hospital Sant Joan de D\u00e9u  Barcelona Spain"},{"name":"Center for the Biomedical Research on Rare Diseases (CIBERER), ISCIII  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"Moreno Escribano","sequence":"additional","affiliation":[{"name":"Neuromuscular Unit, Neurology Department Hospital Cl\u00ednico Universitario Virgen de la Arrixaca  Murcia 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Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carla","family":"Marco","sequence":"additional","affiliation":[{"name":"Motor Neuron Unit, Neurology Department Bellvitge Hospital\u2010IDIBELL  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julita","family":"Medina Castillo","sequence":"additional","affiliation":[{"name":"Physical Medicine and Rehabilitation Department Hospital Sant Joan de Deu  Barcelona Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2349-7481","authenticated-orcid":false,"given":"Nuria","family":"Muelas","sequence":"additional","affiliation":[{"name":"Neuromuscular Unit, Department of Neurology Hospital Universitario y Polit\u00e9cnico la Fe  Valencia Spain"},{"name":"Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Raras (CIBERER)  Valencia 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