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[Choreo-ballistic status treated by GABA].

M Hashimoto, A Yokota, S Matsuoka

    No to Hattatsu = Brain and Development
    |September 1, 1989
    PubMed
    Summary
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    A 9-year-old girl with choreo-ballistic status experienced complete cessation of involuntary movements after intravenous gamma-aminobutyric acid (GABA) therapy. This suggests GABA replacement may treat movement disorders linked to GABA neuron deficiency.

    Area of Science:

    • Neuroscience
    • Neurology
    • Pharmacology

    Background:

    • Chorea and ballism are involuntary movement disorders often linked to basal ganglia dysfunction.
    • Current understanding suggests gamma-aminobutyric acid (GABA) may not cross the blood-brain barrier, limiting its therapeutic use.
    • Previous medical and surgical treatments for choreo-ballistic status in this patient were ineffective.

    Observation:

    • A 9-year-old girl presented with severe choreo-ballistic status.
    • The patient received intravenous gamma-aminobutyric acid (GABA) therapy at maximal doses of 533 mg/kg/day for 3 weeks.

    Findings:

    • Intravenous administration of GABA resulted in the complete cessation of involuntary movements.
    • The treatment was successful despite prior beliefs about GABA's inability to cross the blood-brain barrier.

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    Implications:

    • This case suggests that high-dose intravenous GABA may be a viable therapeutic option for movement disorders caused by GABA neuron hypofunction.
    • Further research is needed to elucidate the mechanism of GABA's intracerebral penetration and confirm its efficacy in similar conditions.
    • This finding opens potential new avenues for treating debilitating hyperkinetic movement disorders.