Segawa disease with a novel heterozygous mutation in exon 5 of the GCH-1 gene (E183K)

Brain Dev. 2009 Feb;31(2):173-5. doi: 10.1016/j.braindev.2008.05.012. Epub 2008 Jul 14.

Abstract

We report a novel missense mutation in the GCH-1 gene resulting in Segawa disease. The patient, a 6-year-old girl, presented with dystonia. Her CSF biopterin and neopterin levels were reduced, suggesting Segawa disease. L-dopa administration led to clinical improvement. Genetic analysis revealed a missense mutation in exon 5 of the GCH-1 gene (E183K). Although dystonia or other movement disorders were not identified in her family, this may be explained by the low penetrance of Segawa disease.

Publication types

  • Case Reports

MeSH terms

  • Base Sequence
  • Biopterins / cerebrospinal fluid
  • Biopterins / metabolism
  • Child
  • Dystonia / drug therapy
  • Dystonia / genetics*
  • Dystonic Disorders / drug therapy
  • Dystonic Disorders / genetics*
  • Exons
  • Female
  • GTP Cyclohydrolase / genetics*
  • Heterozygote
  • Humans
  • Levodopa / therapeutic use
  • Male
  • Mutation, Missense*
  • Neopterin / cerebrospinal fluid
  • Neopterin / metabolism
  • Pedigree
  • Sequence Analysis, DNA

Substances

  • Biopterins
  • Levodopa
  • Neopterin
  • GTP Cyclohydrolase