Anderson-Fabry disease: a protean clinical behavior and a chance diagnosis

J Nephrol. 2005 Nov-Dec;18(6):770-2.

Abstract

Anderson-Fabry disease is a rare inborn X-linked glycosphingolipid storage disorder in which the deficient activity of the enzyme alfa-galactosidase A (alfa-gal A) leads to the progressive tissular accumulation of lipidic molecules which, in turn, cause a protean pattern of multi-organ disfunction. Enzyme replacement therapy has recently become available and has proved to be effective in controlling the disorder. We present and discuss the case of a family with this disease, with special attention to the variability of clinical features and the difficulty of a correct diagnosis.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Biopsy
  • Diagnosis, Differential
  • Echocardiography
  • Electrocardiography
  • Fabry Disease / diagnosis*
  • Fabry Disease / drug therapy
  • Fabry Disease / genetics
  • Humans
  • Isoenzymes / therapeutic use
  • Male
  • Myocardium / pathology
  • Pedigree
  • alpha-Galactosidase / therapeutic use

Substances

  • Isoenzymes
  • alpha-Galactosidase
  • agalsidase beta