Biochemical diagnosis of mucopolysaccharidoses: experience of 297 diagnoses in a 15-year period (1977-1991)

J Inherit Metab Dis. 1993;16(2):473-83. doi: 10.1007/BF00710300.

Abstract

We report the results over 15 years (1977-1991) for biochemical diagnoses of patients referred from throughout Italy and suspected of having a mucopolysaccharidosis. Of these, 147 patients were diagnosed as being homozygous or hemizygous for a specific lysosomal enzyme deficiency; 74 pregnancies at risk were monitored in their families; 76 heterozygote diagnoses were performed on their relatives, with a total of 48 positive diagnoses. We also report the analysis of genomic DNA from 11 unrelated Italian Hunter patients, using pc2S15 probe. DNA from two patients, digested with Pst-I, showed a variant pattern of hybridization caused by deletion or rearrangement of the gene.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Enzymes / deficiency
  • Fibroblasts / enzymology
  • Heterozygote
  • Homozygote
  • Humans
  • Infant
  • Italy
  • Lymphocytes / enzymology
  • Lysosomes / enzymology
  • Mucopolysaccharidoses / diagnosis*
  • Mucopolysaccharidoses / enzymology
  • Mucopolysaccharidoses / genetics
  • Reference Values

Substances

  • Enzymes