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.