SMA CARNI-VAL Trial Part I: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy

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J. Swoboda, Kathryn; B. Scott, Charles; O. Crawford, Thomas; R. Simard, Louise; P. Reyna, Sandra; J. Krosschell, Kristin; Acsadi, Gyula; Elsheik, Bakri; K. Schroth, Mary; D'Anjou, Guy; LaSalle, Bernard; W. Prior, Thomas; L. Sorenson, Susan; Anne Maczulski, Jo; B. Bromberg, Mark; M. Chan, Gary; T. Kissel, John;
  • Publisher: Figshare
  • Related identifiers: doi: 10.1371/journal.pone.0012140
  • Subject: Medicine | Genetics | Neuroscience | sma | carni-val | placebo-controlled | l-carnitine | valproic | spinal | muscular | atrophy

<div><h3>Background</h3><p>Valproic acid (VPA) has demonstrated potential as a therapeutic candidate for spinal muscular atrophy (SMA) <em>in vitro</em> and <em>in vivo</em>.</p><h3>Methods</h3><p>Two cohorts of subjects were enrolled in the SMA CARNIVAL TRIAL, a non-am... View more
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