Juvenile multiple sclerosis-like episodes associated with a defect of mitochondrial beta oxidation

Neurology. 1990 Mar;40(3 Pt 1):487-91. doi: 10.1212/wnl.40.3_part_1.487.

Abstract

We describe a young girl who presented with recurrent episodes of central nervous system (CNS) demyelination mimicking multiple sclerosis. Metabolic evaluations and decreased oxidation of [9,10(n)-3H] palmitate demonstrated defective mitochondrial beta oxidation, but complementation studies of the patient's cells, fused with cell lines with known defects of beta oxidation, failed to identify a known disorder. While progressive CNS demyelination has occurred in patients with defective peroxisomal very long-chain fatty acid oxidation, this is the 1st time it has occurred with defective mitochondrial beta oxidation. This patient appears to represent a novel disorder of beta oxidation producing intermittent demyelination with profound CNS symptoms. Recognition of the defect led to appropriate therapy, which caused marked clinical improvement.

Publication types

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

MeSH terms

  • Amino Acid Metabolism, Inborn Errors / enzymology
  • Child, Preschool
  • Demyelinating Diseases / diagnosis
  • Demyelinating Diseases / metabolism*
  • Diagnosis, Differential
  • Dicarboxylic Acids / urine
  • Fatty Acid Desaturases / metabolism*
  • Female
  • Glutarates / urine
  • Humans
  • Mitochondria / enzymology*
  • Multiple Sclerosis / diagnosis
  • Multiple Sclerosis / metabolism*
  • Oxygen Consumption

Substances

  • Dicarboxylic Acids
  • Glutarates
  • Fatty Acid Desaturases
  • glutaric acid