Phase II open label study of valproic acid in spinal muscular atrophy.

UNLABELLED: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 SMN, supporting the possibility of therapeutic benefit. We performed an open label trial of VPA in 42 subjects with SMA to as...

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Autori principali: Kathryn J Swoboda, Charles B Scott, Sandra P Reyna, Thomas W Prior, Bernard LaSalle, Susan L Sorenson, Janine Wood, Gyula Acsadi, Thomas O Crawford, John T Kissel, Kristin J Krosschell, Guy D'Anjou, Mark B Bromberg, Mary K Schroth, Gary M Chan, Bakri Elsheikh, Louise R Simard
Natura: Artigo
Lingua:Inglês
Pubblicazione: Public Library of Science (PLoS) 2009-01-01
Serie:PLoS ONE
Accesso online:http://europepmc.org/articles/PMC2680034?pdf=render
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