Long-chain 3-hydroxyacyl-coenzyme A dehydrogenase (L-CHAD) deficiency in a patient with the Bannayan-Riley-Ruvalcaba syndrome

Am J Med Genet. 1994 Aug 1;52(1):97-102. doi: 10.1002/ajmg.1320520119.

Abstract

Bannayan-Riley-Ruvalcaba syndrome (BRRS) is an autosomal dominant condition of macrocephaly in combination with lipomas/hemangiomas, hypotonia, developmental delay, and a lipid myopathy. The etiology of the lipid storage myopathy has been unclear. We describe a black boy with findings of BRRS who also has a defect in long-chain fatty acid oxidation expressed in cultured skin fibroblasts as a deficiency of long-chain-L-3-hydroxyacyl-CoA dehydrogenase (L-CHAD). He also has an abnormal brain MRI and increased size of both lower limbs. We present this child because of his unusual combination of findings, and postulate that L-CHAD deficiency may be the cause of the lipid myopathy in BRRS.

Publication types

  • Case Reports

MeSH terms

  • 3-Hydroxyacyl CoA Dehydrogenases / deficiency*
  • Abnormalities, Multiple / enzymology*
  • Abnormalities, Multiple / pathology
  • Hemangioma / pathology
  • Humans
  • Infant
  • Lipoma / pathology
  • Long-Chain-3-Hydroxyacyl-CoA Dehydrogenase
  • Magnetic Resonance Imaging
  • Male
  • Microscopy, Electron
  • Muscles / ultrastructure
  • Muscular Diseases / pathology
  • Soft Tissue Neoplasms / pathology
  • Syndrome

Substances

  • 3-Hydroxyacyl CoA Dehydrogenases
  • Long-Chain-3-Hydroxyacyl-CoA Dehydrogenase