Monitoring of 4-hydroxybutyric acid levels in body fluids during vigabatrin treatment in succinic semialdehyde dehydrogenase deficiency

We report the successful treatment using low‐dose vigabatrin (21.5–34mg/kg/day) of a 10‐year‐old girl with succinic semialdehyde dehydrogenase (SSADH) deficiency We verified that 4‐hydroxybutyric acid (GHB) concentrations in serum, cerebrospinal fluid, and urine continuously decreased in parallel wi...

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Veröffentlicht in:	Annals of neurology 2003-11, Vol.54 (5), p.686-689
Hauptverfasser:	Ergezinger, Katrin, Jeschke, Reinhard, Frauendienst-Egger, Georg, Korall, Herbert, Gibson, K. Michael, Schuster, Volker H.
Format:	Artikel
Sprache:	eng
Schlagworte:	Aldehyde Oxidoreductases - deficiency Biological and medical sciences Child Developmental Disabilities - etiology Enzyme Inhibitors - therapeutic use Female Humans Hydroxybutyrates - analysis Immunomodulators Medical sciences Metabolism, Inborn Errors - drug therapy Metabolism, Inborn Errors - physiopathology Pharmacology. Drug treatments Succinate-Semialdehyde Dehydrogenase Treatment Outcome Vigabatrin - therapeutic use
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