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Published online before print June 14, 2006, doi:10.1212/01.wnl.0000231139.26253.d0)
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NEUROLOGY 2006;67:500-501
© 2006 American Academy of Neurology
Brief Communications

Valproate may improve strength and function in patients with type III/IV spinal muscle atrophy

Conrad C. Weihl, MD, PhD, Anne M. Connolly, MD and Alan Pestronk, MD

From the Department of Neurology, Washington University School of Medicine, St. Louis, MO.

Address correspondence and reprint requests to Dr. A. Pestronk, Department of Neurology, Washington University School of Medicine, 660 South Euclid Avenue, Box 8111, St. Louis, MO 63110; e-mail: pestronka{at}neuro.wustl.edu

Spinal muscular atrophy (SMA) is a recessively inherited motor neuron disease caused by deficient survival motor neuron (SMN) protein. Valproate increases SMN protein in vitro by increasing transcription of SMN2 genes. The authors treated seven adult patients with SMA type III/IV with valproate for a mean duration of 8 months. The treated patients with SMA had increased quantitative muscle strength and subjective function. Further trials of valproate treatment for SMA type III/IV are warranted.

This article was previously published in electronic format as an Expedited E-pub on June 14, 2006, at www.neurology.org.

Disclosure: The authors report no conflicts of interest.

Received January 31, 2006. Accepted in final form March 27, 2006.




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[Abstract] [PDF]


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