Persistent increase of plasma butyryl/isobutyrylcarnitine concentrations as marker of SCAD defect and ethylmalonic encephalopathy

J Inherit Metab Dis. 2006 Oct;29(5):685. doi: 10.1007/s10545-006-0342-8. Epub 2006 Aug 12.

Abstract

High concentrations of butyryl/isobutyrylcarnitine (C(4)-carnitine) in plasma with increase of ethylmalonic acid (EMA) in urine point to different genetic entities, and further investigations are required to differentiate the possible underlying defect. Here we report three unrelated cases, two neurologically affected and one asymptomatic, with this abnormal metabolite pattern due either to mutations in the ETHE1 gene or to a short-chain acyl-CoA dehydrogenase (SCAD) defect.

Publication types

  • Case Reports

MeSH terms

  • Brain Diseases / diagnosis*
  • Butyryl-CoA Dehydrogenase / genetics*
  • Carnitine / analogs & derivatives*
  • Carnitine / blood
  • Humans
  • Malonates / urine
  • Mitochondrial Proteins / genetics
  • Nervous System Diseases
  • Nucleocytoplasmic Transport Proteins / genetics

Substances

  • ETHE1 protein, human
  • Malonates
  • Mitochondrial Proteins
  • Nucleocytoplasmic Transport Proteins
  • isobutyryl-1-carnitine
  • butyrylcarnitine
  • ethylmalonic acid
  • Butyryl-CoA Dehydrogenase
  • Carnitine