Fabry disease: a treatable lysosomal storage disorder

Natl Med J India. 2009 Jan-Feb;22(1):20-2.

Abstract

Fabry disease is a lysosomal storage disease with an X-linked inheritance pattern, which presents in childhood as acroparaesthesias. Its non-specific symptoms often lead to delays in the diagnosis. We report the case of a 13-year-old boy who presented with typical acroparaesthesia of Fabry disease, his younger brother had gastrointestinal manifestations of the disease and their mother's symptoms suggested that she is a carrier. Enzyme replacement therapy helped in ameliorating the patient's symptoms and preventing complications such as renal failure, stroke and cardiovascular disorders.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Fabry Disease / diagnosis
  • Fabry Disease / drug therapy*
  • Fabry Disease / genetics
  • Humans
  • Isoenzymes / therapeutic use*
  • Lysosomal Storage Diseases / diagnosis
  • Lysosomal Storage Diseases / drug therapy
  • Lysosomal Storage Diseases / genetics
  • Male
  • Recombinant Proteins / therapeutic use*
  • Risk Factors
  • alpha-Galactosidase / metabolism*
  • alpha-Galactosidase / therapeutic use

Substances

  • Isoenzymes
  • Recombinant Proteins
  • alpha-Galactosidase
  • agalsidase beta