Peripheral neuropathy in a patient with D-2-hydroxyglutaric aciduria

J Inherit Metab Dis. 2009 Dec:32 Suppl 1:S21-5. doi: 10.1007/s10545-009-0933-2. Epub 2009 Jan 26.

Abstract

D-2-hydroxyglutaric aciduria (D-2-HGA; OMIM 600721) is a rare autosomal recessive neurometabolic disorder with a wide clinical spectrum. The severe phenotype is homogeneous and is characterized by early infantile-onset epileptic encephalopathy with hypotonia, delayed cerebral visual development, cardiomyopathy and facial dysmorphic features. The mild phenotype has a more variable clinical expression with hypotonia and developmental delay. We present peripheral neuropathy as an additional clinical and electrophysiological feature in a 16-year-old boy with a homozygous missense mutation in exon 3 of the D-2-hydroxyglutarate dehydrogenase gene (D2HGDH) at position c.458T>C. This mutation results in replacement of a methionine residue, which was highly conserved during evolution, by threonine (p.Met153Thr).

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Alcohol Oxidoreductases / genetics*
  • Brain / pathology
  • Brain Diseases, Metabolic, Inborn / complications*
  • Brain Diseases, Metabolic, Inborn / enzymology
  • Brain Diseases, Metabolic, Inborn / genetics
  • Brain Diseases, Metabolic, Inborn / pathology
  • Electrophysiological Phenomena
  • Genes, Recessive
  • Homozygote
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Mutation, Missense
  • Neural Conduction / genetics
  • Peripheral Nervous System Diseases / etiology*
  • Peripheral Nervous System Diseases / genetics
  • Peripheral Nervous System Diseases / physiopathology
  • Phenotype

Substances

  • Alcohol Oxidoreductases
  • D2HGDH protein, human

Supplementary concepts

  • 2-Hydroxyglutaricaciduria