Tyrosine hydroxylase deficiency with severe clinical course: clinical and biochemical investigations and optimization of therapy.

Dionisi-Vici, C; Hoffmann, G F; Leuzzi, V; Hoffken, H; Bräutigam, C; Rizzo, C; Steebergen-Spanjers, G C; Smeitink, J A et al. · J Pediatr · 2000

case_report · Level V

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Abstract

Tyrosine hydroxylase deficiency was diagnosed after determination of cerebrospinal fluid neurotransmitters and DNA analysis in a child with severe axial hypotonia and hypokinesia associated with dystonic and ballistic movements. L-dopa therapy was unsuccessful, whereas a combination with selegiline, a selective monoamine oxidase-beta inhibitor, with low-dose L-dopa markedly improved the severe clinical picture.

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