γ-乙烯基γ-氨基丁酸在弗里德赖希共济失调中的作用。

Effect of gamma-vinyl GABA in Friedreich's ataxia.

作者信息

De Smet Y, Mear J Y, Tell G, Schechter P H, Lhermitte F, Agid Y

出版信息

Can J Neurol Sci. 1982 May;9(2):171-3. doi: 10.1017/s0317167100043894.

Abstract

Gamma-Vinyl GABA, an irreversible inhibitor of GABA-transaminase, was administered orally in two daily doses of 250 mg to 10 patients with cerebellar ataxia (9 with Friedreich's ataxia, one with olivo-ponto-cerebellar atrophy) for at least one month in an open study. No significant difference occurred in the disability scores of cerebellar symptomatology for the group as a whole, but seven patients showed some improvement in scores with treatment and two patients claimed marked subjective amelioration. Tolerance to Gamma-Vinyl-GABA treatment was excellent. These preliminary results suggest that further studies with well-tolerated agents which enhance CNS GABA-ergic function are warranted in patients with cerebellar ataxia.

摘要

γ-乙烯基氨基丁酸是一种γ-氨基丁酸转氨酶的不可逆抑制剂，在一项开放性研究中，以每日两次、每次250毫克的剂量口服给予10例小脑性共济失调患者（9例弗里德赖希共济失调患者，1例橄榄体脑桥小脑萎缩患者），治疗至少1个月。整个组的小脑症状残疾评分没有显著差异，但7例患者在治疗后评分有所改善，2例患者称主观症状有明显改善。对γ-乙烯基氨基丁酸治疗的耐受性良好。这些初步结果表明，对于小脑性共济失调患者，有必要进一步研究耐受性良好且能增强中枢神经系统γ-氨基丁酸能功能的药物。