[Morquio A disease: clinical and molecular study of Tunisian patients]

Ann Biol Clin (Paris). 2011 Jul-Aug;69(4):425-9. doi: 10.1684/abc.2011.0593.
[Article in French]

Abstract

Type IVA mucopolysaccharidosis or Morquio A disease is a lysosomal storage disease, autosomal recessive, caused by deficiency of the N-acetylgalactosamine-6-sulfate sulfatase or GALNS. The severe phenotype is characterized by a severe skeletal dysplasia without any mental retardation. The aim of this study was to propose a strategy of molecular and prenatal diagnosis of this pathology. A molecular study was carried out on 7 patients MPS IVA issued from 5 unrelated families recruited from different Tunisian regions. All the patients were offspring of consanguineous marriages. The clinical and biologic study confirmed the diagnosis of MPS IVA within the 7 studied patients. Three GALNS mutations were identified by molecular analysis: IVS1+1G>A, G66R and A85T. The unions between Tunisian relatives are important and increase the Morquio A incidence in Tunisia. The identification of GALNS mutations in the Tunisian population permits better understanding of the Morquio A phenotype, a reliable genetic counselling and a molecular prenatal diagnosis to Tunisian at-risk relatives.

Publication types

  • English Abstract

MeSH terms

  • Adolescent
  • Biomarkers / metabolism
  • Child
  • Chondroitinsulfatases / genetics*
  • Consanguinity
  • Exons
  • Female
  • Genetic Counseling
  • Humans
  • Male
  • Mucopolysaccharidosis IV / diagnosis*
  • Mucopolysaccharidosis IV / epidemiology
  • Mucopolysaccharidosis IV / genetics*
  • Mutation*
  • Phenotype
  • Polymorphism, Genetic
  • Prenatal Diagnosis
  • Tunisia / epidemiology
  • Young Adult

Substances

  • Biomarkers
  • Chondroitinsulfatases
  • GALNS protein, human