Journal
NEUROLOGY
Volume 67, Issue 3, Pages 500-501Publisher
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1212/01.wnl.0000231139.26253.d0
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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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