[Management of Fabry disease]

Orv Hetil. 2010 Aug 1;151(31):1243-51. doi: 10.1556/OH.2010.28796.
[Article in Hungarian]

Abstract

Fabry disease is a rare, X-linked lysosomal storage disorder that leads to accumulation of globotriaosylceramide in different tissues of the body. The disease is progressive and the first symptoms usually present in childhood. Consequences of the disease are disability and premature death. The disease in females could be as severe as in males although women may be asymptomatic. The possibility of enzyme replacement therapy has made it necessary to elaborate a comprehensive guideline for the diagnosis and treatment follow-up. The guideline has been summarized by a Hungarian multi-disciplinary working group consisting of physicians who are involved in diagnosis and care of Fabry patients. Previous clinical studies, published articles, and recently established international treatment guidelines were reviewed by the group.

Publication types

  • English Abstract
  • Practice Guideline
  • Review

MeSH terms

  • Clinical Trials as Topic
  • Enzyme Replacement Therapy
  • Fabry Disease* / complications
  • Fabry Disease* / diagnosis
  • Fabry Disease* / drug therapy
  • Fabry Disease* / enzymology
  • Fabry Disease* / physiopathology
  • Female
  • Heterozygote
  • Humans
  • Male
  • Treatment Outcome
  • alpha-Galactosidase / genetics
  • alpha-Galactosidase / therapeutic use*

Substances

  • alpha-Galactosidase