3-Methylglutaconic aciduria type I: clinical heterogeneity as a neurometabolic disease

J Inherit Metab Dis. 1999 Feb;22(1):1-8. doi: 10.1023/a:1005421111554.

Abstract

3-Methylglutaconic (3-MGC) aciduria with 3-methylglutaconyl-CoA hydratase deficiency (3-MGC aciduria type I) is a rare inherited metabolic disease of L-leucine catabolism. We describe a 9-month-old Japanese boy with this disorder who showed progressive neurological impairments presented as quadriplegia, athetoid movements and severe psychomotor retardation from 4 months of age. This finding indicates the existence of clinical heterogeneity in 3-MGC aciduria type I, suggesting it may present as a neurometabolic disease.

Publication types

  • Case Reports

MeSH terms

  • Acidosis, Renal Tubular / complications*
  • Acidosis, Renal Tubular / metabolism
  • Acidosis, Renal Tubular / physiopathology
  • Acidosis, Renal Tubular / urine
  • Athetosis / etiology
  • Glutarates / urine*
  • Humans
  • Infant
  • Male
  • Nervous System Diseases / etiology*
  • Psychomotor Disorders / etiology
  • Quadriplegia / etiology

Substances

  • Glutarates
  • 3-methylglutaconic acid