Valproate may improve strength and function in patients with type III/IV spinal muscle atrophy
Valproate may improve strength and function in patients with type III/IV spinal muscle atrophy
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DOI：
10.1212/01.wnl.0000231139.26253.d0
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发表时间：
2006-08-08
期刊：
影响因子：
9.9
通讯作者：
Pestronk, Alan
中科院分区：
文献类型：
--
作者：
Weihl, Conrad C.;Connolly, Anne M.;Pestronk, Alan
脊髓性肌萎缩症(SMA)是由运动神经元存活蛋白(SMN)缺乏引起的一种隐性遗传性运动神经元疾病。丙戊酸盐在体外通过增加SMN2基因的转录增加SMN蛋白。作者用丙戊酸盐治疗7例成人SMA III/IV型患者，平均疗程8个月。治疗后SMA患者的定量肌力和主观功能均有提高。丙戊酸盐治疗SMA III/IV型的进一步试验是有必要的。
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.