{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,28]],"date-time":"2026-06-28T14:14:18Z","timestamp":1782656058110,"version":"3.54.5"},"reference-count":30,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2019,12,11]],"date-time":"2019-12-11T00:00:00Z","timestamp":1576022400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"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":["Muscle and Nerve"],"published-print":{"date-parts":[[2020,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>With the advent of disease\u2010altering therapies for spinal muscular atrophy (SMA), there is a requirement to better characterize outcome measures, particularly in milder forms of disease.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Maximal voluntary isometric contraction testing and 6\u2010minute walk test (6MWT) performed in ambulatory SMA adults as part of the SMA\u2010VALIANT trial were analyzed. Test\u2013retest reliability and correlation with other candidate biomarkers and outcomes were investigated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Maximal voluntary isometric contraction testing and 6MWT showed good test\u2013retest reliability (intraclass correlation coefficient = 0.98 and 0.85, respectively). Maximal voluntary isometric contraction testing and 6MWT demonstrated very strong correlation (<jats:italic>r<\/jats:italic> = 0.83, <jats:italic>P<\/jats:italic> &lt;. 0001), and each correlated with the SMA Functional Rating Scale (<jats:italic>r<\/jats:italic> = 0.7, <jats:italic>P<\/jats:italic>\u2009&lt;\u2009.0001 and <jats:italic>r<\/jats:italic> = 0.65, <jats:italic>P<\/jats:italic> = .0001, respectively), lean muscle mass (<jats:italic>r<\/jats:italic> = 0.68, <jats:italic>P<\/jats:italic>\u2009&lt;\u2009.0001 and <jats:italic>r<\/jats:italic> = 0.56, <jats:italic>P<\/jats:italic> = .001, respectively), and ulnar compound muscle action potential (<jats:italic>r<\/jats:italic> = 0.57, <jats:italic>P<\/jats:italic> = .0008 and <jats:italic>r<\/jats:italic> = 0.47, <jats:italic>P<\/jats:italic> = .008, respectively).<\/jats:p><\/jats:sec><jats:sec><jats:title>Discussion<\/jats:title><jats:p>Maximal voluntary isometric contraction testing and 6MWT are suitable outcomes for use in ambulatory adults with SMA. Maximal voluntary isometric contraction testing may be preferable because of superior test\u2013retest reliability and closer associations with other outcomes and biomarkers of neuromuscular function.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.26756","type":"journal-article","created":{"date-parts":[[2019,11,14]],"date-time":"2019-11-14T17:23:07Z","timestamp":1573752187000},"page":"187-191","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Outcome measures in a cohort of ambulatory adults with spinal muscular atrophy"],"prefix":"10.1002","volume":"61","author":[{"given":"Bakri","family":"Elsheikh","sequence":"first","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine The Ohio State University Wexner Medical Center  Columbus Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wendy","family":"King","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine The Ohio State University Wexner Medical Center  Columbus Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan","family":"Peng","sequence":"additional","affiliation":[{"name":"Department of Biomedical Informatics, Center for Biostatistics The Ohio State University Wexner Medical Center  Columbus Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4593-6342","authenticated-orcid":false,"given":"Kathy J.","family":"Swoboda","sequence":"additional","affiliation":[{"name":"Department of Neurology Massachusetts General Hospital  Boston Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra P.","family":"Reyna","sequence":"additional","affiliation":[{"name":"Biogen  Cambridge Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernard","family":"LaSalle","sequence":"additional","affiliation":[{"name":"Department of Biomedical Informatics University of Utah School of Medicine  Salt Lake City Utah"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas W.","family":"Prior","sequence":"additional","affiliation":[{"name":"Department of Molecular Pathology The Ohio State University Wexner Medical Center  Columbus Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"W. 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