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. 2006 Aug 8;67(3):500-1.
doi: 10.1212/01.wnl.0000231139.26253.d0. Epub 2006 Jun 14.

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

Conrad C Weihl  1 , Anne M Connolly, Alan Pestronk
Affiliations

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

Conrad C Weihl et al. Neurology. .

Abstract

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.

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