Interdisciplinary approach towards female patients with Fabry disease

Eur J Clin Invest. 2012 Apr;42(4):455-62. doi: 10.1111/j.1365-2362.2011.02614.x. Epub 2011 Nov 3.

Abstract

Background: Fabry disease (FD) is a rare X-linked lysosomal storage disorder leading to an accumulation of globotriaosylceramides in the lysosomes of various organs.

Design: Being X-chromosomal-linked, most studies in the past have focused on involvement in male patients. However, it has been elucidated recently that female patients can present typical organ involvement and thus need to be treated, respectively.

Conclusions: This review wants to give a systematical overview of the typical organ involvement in female patients with FD. Moreover, therapy recommendations especially for female patients are discussed.

Publication types

  • Case Reports
  • Review

MeSH terms

  • Adult
  • Brain Diseases / diagnosis*
  • Brain Diseases / genetics
  • Brain Diseases / metabolism
  • Cardiomyopathies / diagnosis*
  • Cardiomyopathies / genetics
  • Cardiomyopathies / metabolism
  • Fabry Disease / diagnosis*
  • Fabry Disease / genetics
  • Fabry Disease / metabolism
  • Female
  • Glycosphingolipids / metabolism
  • Humans
  • Kidney Diseases / diagnosis*
  • Kidney Diseases / genetics
  • Kidney Diseases / metabolism
  • Middle Aged
  • Patient Care Team
  • Severity of Illness Index
  • Trihexosylceramides / metabolism
  • Young Adult
  • alpha-Galactosidase / metabolism

Substances

  • Glycosphingolipids
  • Trihexosylceramides
  • globotriaosylceramide
  • alpha-Galactosidase