Phase II Open Label Study of Valproic Acid in Spinal Muscular Atrophy

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J. Swoboda, Kathryn; B. Scott, Charles; P. Reyna, Sandra; W. Prior, Thomas; LaSalle, Bernard; L. Sorenson, Susan; Wood, Janine; Acsadi, Gyula; O. Crawford, Thomas; T. Kissel, John; J. Krosschell, Kristin; D'Anjou, Guy; B. Bromberg, Mark; K. Schroth, Mary; M. Chan, Gary; Elsheikh, Bakri; R. Simard, Louise;

<div><h3></h3><p>Preliminary in vitro and in vivo studies with valproic acid (VPA) in cell lines and patients with spinal muscular atrophy (SMA) demonstrate increased expression of <em>SMN</em>, supporting the possibility of therapeutic benefit. We performed an open lab... View more
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