Recurrence of the D409H mutation in Spanish Gaucher disease patients: description of a new homozygous patient and haplotype analysis

J Med Genet. 1998 Sep;35(9):775-7. doi: 10.1136/jmg.35.9.775.

Abstract

Gaucher disease results, in most patients, from mutations in the gene encoding glucocerebrosidase. Mutation D409H is the third most frequent in Spanish patients, accounting for 5.7% of all mutated alleles. This allele is associated mainly with the neurological forms of the disease. Recently, homozygosity for the D409H mutation has been associated with a particular phenotype, including specific cardiovascular symptoms. Here we report a second Spanish patient bearing the D409H/D409H genotype with a very early manifestation of the disease. The patient started enzyme replacement therapy at 3 months of age. A common origin for the Spanish D409H alleles was ruled out by haplotype analysis using an internal polymorphism of the glucocerebrosidase gene and two external microsatellite markers.

Publication types

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

MeSH terms

  • Gaucher Disease / drug therapy
  • Gaucher Disease / genetics*
  • Genetic Markers
  • Glucosylceramidase / genetics
  • Glucosylceramidase / therapeutic use
  • Haplotypes*
  • Homozygote*
  • Humans
  • Infant, Newborn
  • Point Mutation / genetics*
  • Polymorphism, Single-Stranded Conformational
  • Spain

Substances

  • Genetic Markers
  • alglucerase
  • Glucosylceramidase