Effect of gamma-vinyl GABA in Friedreich's ataxia.
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.
D000612:4-Aminobutyrate Transaminase / Q000037:antagonists & inhibitors*; D000293:Adolescent; D000328:Adult; D000614:Aminocaproates / Q000627:therapeutic use*; D004361:Drug Tolerance; D005260:Female; D005621:Friedreich Ataxia / Q000188:drug therapy*; D006801:Humans; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D000637:Transaminases / Q000037:antagonists & inhibitors*; D020888:Vigabatrin; D005680:gamma-Aminobutyric Acid / Q000378:metabolism
D000614:Aminocaproates; D005680:gamma-Aminobutyric Acid; D000637:Transaminases; D000612:4-Aminobutyrate Transaminase; D020888:Vigabatrin