{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T03:10:38Z","timestamp":1791169838469,"version":"4.1.0"},"reference-count":34,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2013,6,13]],"date-time":"2013-06-13T00:00:00Z","timestamp":1371081600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"Families of Spinal Muscular Atrophy and also by grants from the Center for Clinical and Translational Sciences, University of Utah","award":["UL1RR025764"],"award-info":[{"award-number":["UL1RR025764"]}]},{"name":"Center for Clinical and Translational Sciences, Ohio State University","award":["UL1RR025755"],"award-info":[{"award-number":["UL1RR025755"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2014,2]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:p><jats:italic>Introduction<\/jats:italic>: An open\u2010label trial suggested that valproic acid (VPA) improved strength in adults with spinal muscular atrophy (SMA). We report a 12\u2010month, double\u2010blind, cross\u2010over study of VPA in ambulatory SMA adults. <jats:italic>Methods<\/jats:italic>: There were 33 subjects, aged 20\u201355 years, included in this investigation. After baseline assessment, subjects were randomized to receive VPA (10\u201320 mg\/kg\/day) or placebo. At 6 months, patients were switched to the other group. Assessments were performed at 3, 6, and 12 months. The primary outcome was the 6\u2010month change in maximum voluntary isometric contraction testing with pulmonary, electrophysiological, and functional secondary outcomes. <jats:italic>Results<\/jats:italic>: Thirty subjects completed the study. VPA was well tolerated, and compliance was good. There was no change in primary or secondary outcomes at 6 or 12 months. <jats:italic>Conclusions<\/jats:italic>: VPA did not improve strength or function in SMA adults. The outcomes used are feasible and reliable and can be employed in future trials in SMA adults. <jats:italic>Muscle Nerve<\/jats:italic> <jats:bold>49<\/jats:bold>: 187\u2013192, 2014<\/jats:p>","DOI":"10.1002\/mus.23904","type":"journal-article","created":{"date-parts":[[2013,5,16]],"date-time":"2013-05-16T12:28:57Z","timestamp":1368707337000},"page":"187-192","source":"Crossref","is-referenced-by-count":69,"title":["SMA valiant trial: A prospective, double\u2010blind, placebo\u2010controlled trial of valproic acid in ambulatory adults with spinal muscular atrophy"],"prefix":"10.1002","volume":"49","author":[{"given":"John T.","family":"Kissel","sequence":"first","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine Ohio State University, Wexner Medical Center 395 West 12th Avenue Columbus Ohio 43210 USA"},{"name":"Department of Pediatrics Ohio State University Columbus Ohio USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bakri","family":"Elsheikh","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine Ohio State University, Wexner Medical Center 395 West 12th Avenue Columbus Ohio 43210 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wendy M.","family":"King","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine Ohio State University, Wexner Medical Center 395 West 12th Avenue Columbus Ohio 43210 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miriam","family":"Freimer","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine Ohio State University, Wexner Medical Center 395 West 12th Avenue Columbus Ohio 43210 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Charles B.","family":"Scott","sequence":"additional","affiliation":[{"name":"CBS Squared, Inc. Fort Washington  Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stephen J.","family":"Kolb","sequence":"additional","affiliation":[{"name":"Department of Neurology, Division of Neuromuscular Medicine Ohio State University, Wexner Medical Center 395 West 12th Avenue Columbus Ohio 43210 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sandra P.","family":"Reyna","sequence":"additional","affiliation":[{"name":"Department of Neurology University of Utah School of Medicine Salt Lake City Utah USA"},{"name":"Department of Pediatrics University of Utah School of Medicine Salt Lake City Utah USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas O.","family":"Crawford","sequence":"additional","affiliation":[{"name":"Department of Neurology Johns Hopkins University School of Medicine Baltimore Maryland USA"},{"name":"Department of Pediatrics Johns Hopkins University School of Medicine Baltimore Maryland 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