[Pediatric aspects of Fabry's disease]

Arch Pediatr. 2007 Jul;14(7):909-14. doi: 10.1016/j.arcped.2007.02.090. Epub 2007 Apr 24.
[Article in French]

Abstract

Fabry's disease is a rare X-linked inborn error of glycosphingolipid metabolism characterised by an abnormal lipid storage due to a defect of lysozomal alphagalactosidase. The consequence is a storage of glycosphingolipides in all tissues. This storage in vessels's endothelial cells is responsible, in males, for severe ischemic lesions leading to progressive kidney failure, cardiac and cerebral dysfunctions. Similarly, it involves ocular tissues, mainly the cornea, the conjunctiva and the lens. The corneal storage, known as cornea verticillata, is a clinical marker easy to recognize by slit lamp examination of the affected males and carrier females. The enzymatic activity of alphagalactosidase is reduced in tears. Characteristic lamellar bodies can be observed by electron microscopy study of a conjunctival biopsy. Until recently, treatment was limited to symptomatic management of pain, and end-stage complications of renal failure, cardiac or brain disease. Recent studies have demonstrated that enzyme replacement therapy by genetic engineering is now shown to be promising for affected patients.

Publication types

  • English Abstract
  • Review

MeSH terms

  • Child
  • Eye Diseases / etiology
  • Fabry Disease* / complications
  • Fabry Disease* / diagnosis
  • Fabry Disease* / genetics
  • Fabry Disease* / therapy
  • Female
  • Humans
  • Male
  • Pedigree