Article
Therapeutic intervention in mice deficient for succinate semialdehyde dehydrogenase (gamma-hydroxybutyric aciduria).
The Journal of pharmacology and experimental therapeutics - 1 Jul 2002
Gupta Maneesh, Greven Rachel, Jansen Erwin E W, Jakobs Cornelis, Hogema Boris M, Froestl Wolfgang, Snead O Carter, Bartels Hilke, Grompe Markus, Gibson K Michael
Abstract excerpt
Therapeutic intervention for human succinic semialdehyde dehydrogenase (SSADH) deficiency (gamma-hydroxybutyric aciduria) has been limited to vigabatrin (VGB). Pharmacologically, VGB should be highly effective due to 4-aminobutyrate-transaminase (GABA-transaminase) inhibition, lowering succinic semialdehyde and, thereby, gamma-hydroxybutyric acid (GHB) levels. Unfortunately, clinical efficacy has been limited....
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