Dopa-responsive infantile hypokinetic rigid syndrome due to dominant guanosine triphosphate cyclohydrolase 1 deficiency

J Neurol Sci. 2007 May 15;256(1-2):90-3. doi: 10.1016/j.jns.2007.02.007. Epub 2007 Mar 21.

Abstract

We report on a GTP cyclohydrolase 1 mutation-confirmed heterozygous case presenting with an infantile hypokinetic rigid syndrome and delay in attainment of motor milestones starting from the first year of life. He had a family history of dopa-responsive dystonia-parkinsonism. CSF neopterin, biopterin and HVA values were decreased. Molecular study of GCH-1 gene showed the Q89X mutation in exon 1. Treatment with l-dopa resulted in a complete remission of symptoms.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antiparkinson Agents / adverse effects*
  • Family Health
  • Female
  • GTP Cyclohydrolase / deficiency*
  • Humans
  • Hypokinesia / chemically induced*
  • Hypokinesia / physiopathology
  • Infant
  • Levodopa / adverse effects*
  • Male
  • Muscle Rigidity*
  • Retrospective Studies

Substances

  • Antiparkinson Agents
  • Levodopa
  • GTP Cyclohydrolase