Treatment of sodium valproate and amantazine hydrochloride in spinal muscular atrophy

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Spinal muscular atrophy (SMA) is characterized by degeneration of the motoric neurons of the anterior horn cells of the spinal cord, leading to muscle atrophydue to mutation of telomeric survival motor neuron (SMN1) located in chromosome on 5q13. SMN2, high homology of SMN1, is determined to disease severity. SMA II and III have 3 or 4 numbers of SMN2 gene. Recently, sodium valproate (VPA) treated with epilepsy, increased number of SMN2 gene in vitro. Increased muscle strength with Amantazine treatment has reported in SMA II patients. We studied effect of treatment with VPA and amantazine using ccultured skin fibroblasts derived from SMA patints based on SMN mRNA and protein levels. Results: VPA treatment was effective but Amantazine was no effeitve in vitro.