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

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 assess safety...

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Detalles Bibliográficos
Autores principales: Swoboda, Kathryn J., Scott, Charles B., Reyna, Sandra P., Prior, Thomas W., LaSalle, Bernard, Sorenson, Susan L., Wood, Janine, Acsadi, Gyula, Crawford, Thomas O., Kissel, John T., Krosschell, Kristin J., D'Anjou, Guy, Bromberg, Mark B., Schroth, Mary K., Chan, Gary M., Elsheikh, Bakri, Simard, Louise R.
Formato: Texto
Lenguaje:English
Publicado: Public Library of Science 2009
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2680034/
https://www.ncbi.nlm.nih.gov/pubmed/19440247
http://dx.doi.org/10.1371/journal.pone.0005268

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2680034/
https://www.ncbi.nlm.nih.gov/pubmed/19440247
http://dx.doi.org/10.1371/journal.pone.0005268

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