{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T23:54:24Z","timestamp":1791244464482,"version":"4.1.0"},"reference-count":34,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2021,6,24]],"date-time":"2021-06-24T00:00:00Z","timestamp":1624492800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100002426","name":"Fondazione Telethon","doi-asserted-by":"publisher","award":["GSP 13002"],"award-info":[{"award-number":["GSP 13002"]}],"id":[{"id":"10.13039\/501100002426","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100011708","name":"SMA Trust","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100011708","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000894","name":"Spinal Muscular Atrophy Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000894","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007721","name":"Cure SMA","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007721","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Ann Clin Transl Neurol"],"published-print":{"date-parts":[[2021,8]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objective<\/jats:title><jats:p>We report longitudinal data from 144 type III SMA pediatric and adult patients treated with nusinersen as part of an international effort.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Patients were assessed using Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM), and 6\u2010Minute Walk Test (6MWT) with a mean follow\u2010up of 1.83\u00a0years after nusinersen treatment.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Over 75% of the 144 patients had a 12\u2010month follow\u2010up. There was an increase in the mean scores from baseline to 12\u00a0months on both HFMSE (1.18 points, <jats:italic>p<\/jats:italic>\u00a0=\u00a00.004) and RULM scores (0.58 points, <jats:italic>p<\/jats:italic>\u00a0=\u00a00.014) but not on the 6MWT (mean difference\u00a0=\u00a06.65\u00a0m, <jats:italic>p<\/jats:italic>\u00a0=\u00a00.33).<\/jats:p><jats:p>When the 12\u2010month HFMSE changes in the treated cohort were compared to an external cohort of untreated patients, in all untreated patients older than 7\u00a0years, the mean changes were always negative, while always positive in the treated ones. To reduce a selection bias, we also used a multivariable analysis. On the HFMSE scale, age, gender, baseline value, and functional status contributed significantly to the changes, while the number of SMN2 copies did not contribute. The effect of these variables was less obvious on the RULM and 6MWT.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>Our results expand the available data on the effect of Nusinersen on type III patients, so far mostly limited to data from adult type III patients.<\/jats:p><\/jats:sec>","DOI":"10.1002\/acn3.51411","type":"journal-article","created":{"date-parts":[[2021,6,24]],"date-time":"2021-06-24T12:47:48Z","timestamp":1624538868000},"page":"1622-1634","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":48,"title":["Nusinersen in pediatric and adult patients with type III spinal muscular atrophy"],"prefix":"10.1002","volume":"8","author":[{"given":"Maria Carmela","family":"Pera","sequence":"first","affiliation":[{"name":"Pediatric Neurology Universit\u00e0 Cattolica del Sacro Cuore Rome Italy"},{"name":"Centro Clinico Nemo Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome 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