Brain dysgenesis and congenital intracerebral calcification associated with 3-hydroxyisobutyric aciduria

J Pediatr. 1992 Jul;121(1):86-9. doi: 10.1016/s0022-3476(05)82549-1.

Abstract

Monozygotic male twins born to nonconsanguineous parents had dysmorphic facial features, microcephaly, migrational brain disorder, and congenital intracerebral calcification. They excreted excessive amounts of 3-hydroxyisobutyric acid, a metabolite of valine, and had evidence of impaired oxidative metabolism and metabolic acidosis. The level of 3-hydroxyisobutyrate in stored samples of midtrimester amniotic fluid was found to be high. The association of 3-hydroxyisobutyric aciduria with brain dysgenesis is a newly recognized mendelian disorder; its recurrence in a family at risk is potentially avoidable by prenatal diagnosis.

Publication types

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

MeSH terms

  • Amniotic Fluid / chemistry
  • Brain / abnormalities*
  • Brain Diseases / congenital*
  • Brain Diseases / genetics
  • Calcinosis / congenital*
  • Calcinosis / genetics
  • Cytogenetics
  • DNA Fingerprinting
  • Diseases in Twins*
  • Humans
  • Hydroxybutyrates / analysis
  • Hydroxybutyrates / urine*
  • Infant, Newborn
  • Male
  • Metabolism, Inborn Errors / genetics
  • Polymerase Chain Reaction
  • Twins, Monozygotic*

Substances

  • Hydroxybutyrates
  • 3-hydroxyisobutyric acid