Valproate may improve strength and function in patients with type III/IV spinal muscle atrophy.
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.
Weihl
Conrad C
CC
Department of Neurology, Washington University School of Medicine, 660 South Euclid Avenue, Box 8111, St. Louis, MO 63110, USA.
Connolly
Anne M
AM
Pestronk
Alan
A
D000293:Adolescent; D000328:Adult; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009046:Motor Neurons / Q000187:drug effects*; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy*; D012189:Retrospective Studies; D014635:Valproic Acid / Q000494:pharmacology / Q000627:therapeutic use*
D014635:Valproic Acid