Clinical heterogeneity in Sanfilippo disease (mucopolysaccharidosis III) type D: presentation of two new cases

Eur J Pediatr. 1983 Apr;140(2):130-3. doi: 10.1007/BF00441662.

Abstract

Clinical, radiological and biochemical findings of two new cases of Sanfilippo disease, type D are reported. A high percentage of heparan sulfate was found in the urinary glycosaminoglycan pattern and a severe deficiency of N-acetylglucosamine-6-sulfate sulfatase was demonstrated in skin cultured fibroblasts from the patients. One of the patients presented mild intellectual impairment which differentiates him from the other cases described to date.

Publication types

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

MeSH terms

  • Child
  • Child, Preschool
  • Female
  • Fibroblasts / enzymology
  • Glycosaminoglycans / urine
  • Heparitin Sulfate / analysis
  • Humans
  • Male
  • Mucopolysaccharidoses / diagnosis*
  • Mucopolysaccharidosis III / diagnosis*
  • Sulfatases / analysis

Substances

  • Glycosaminoglycans
  • Heparitin Sulfate
  • Sulfatases
  • N-acetylglucosamine-6-sulfatase