{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,20]],"date-time":"2025-10-20T18:58:30Z","timestamp":1760986710648,"version":"3.30.2"},"reference-count":55,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2024,10,3]],"date-time":"2024-10-03T00:00:00Z","timestamp":1727913600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2024,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction\/Aims<\/jats:title><jats:p>Persons with spinal muscular atrophy (pwSMA) report progressive muscle weakness but also reduced endurance when performing repetitive tasks in daily life, referred to as \u201cperformance fatigability\u201d (PF). Data regarding the effects of the new disease\u2010modifying drugs on PF are scarce. Thus, our main objective was to examine PF in adult ambulatory pwSMA treated long\u2010term with nusinersen.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Six\u2010minute walk test (6MWT) data from 14 adult pwSMA treated with nusinersen for up to 70\u2009months were retrospectively analyzed to determine PF. Performance fatigability was defined as the percentage change in the distance covered between the last and first minute of the 6MWT. In addition, relationships between PF and other clinical features were assessed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Performance fatigability was found in 12\/14 pwSMA (85.7%) prior to treatment. The mean distance walked in the sixth minute (71.1\u2009m) was shorter than the distance covered in the first minute (81.8\u2009m), corresponding to a mean PF of 13.1% (95% confidence interval (CI): 6.5\u201319.6, <jats:italic>p<\/jats:italic>\u2009=\u2009.0007). During treatment with nusinersen, there was a mean reduction in PF of 5.6% (95% CI: \u221210.0 to \u22121.3, <jats:italic>p<\/jats:italic>\u2009=\u2009.0148). We found no relationship between PF and fatigue as measured by the Fatigue Severity Scale.<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>This study demonstrates the presence of PF as an independent component of motor impairment and as a potential therapeutic target in our cohort of adult ambulatory pwSMA. Furthermore, the observations in our cohort suggest that nusinersen may have a beneficial effect on PF.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.28269","type":"journal-article","created":{"date-parts":[[2024,10,3]],"date-time":"2024-10-03T11:13:38Z","timestamp":1727954018000},"page":"1223-1229","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Performance fatigability in adults with spinal muscular atrophy treated long\u2010term with nusinersen"],"prefix":"10.1002","volume":"70","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2774-067X","authenticated-orcid":false,"given":"Benjamin","family":"Stolte","sequence":"first","affiliation":[{"name":"Department of Neurology and Center for Translational Neuro\u2010 and Behavioral Sciences (C\u2010TNBS) University Hospital Essen  Essen 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