Summary
Fabry disease is a rare, X-linked lysosomal storage disease caused by an inborn deficiency of α-galactosidase A, which results in the progressive accumulation of globotriaosylceramide and other neutral glycolipids in a range of cells and tissues. In association with the renal and cardiac insufficiency, cerebrovascular complications can result in the death of the patients. Several mechanisms causing vascular damage that leads to the development of deep-white matter lesions have been described. Recent clinical trials strongly suggest that statins protect against stroke by neuroprotective properties or pleiotropic effects. Aim: To evaluate evidence and potential beneficial effects of statins in the vasculopathy of Fabry disease.
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Abbreviations
- ERT:
-
enzyme replacement therapy
- FD:
-
Fabry disease
- Gb3 :
-
globotriaosylceramide
- Gla:
-
α-galactosidase A
- HMG-CoA:
-
3-hydroxy-3-methylglutaryl coenzyme A
- ICAM-1:
-
intercellular adhesion molecule-1
- IMT:
-
intima-media thickness
- NOS:
-
nitric oxide synthase
- PAI-1:
-
plasminogen activator inhibitor-1
- VCAM-1:
-
vascular cell adhesion molecule-1
- WML:
-
white-matter lesion
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Communicating editor: Frits Wijburg
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Politei, J.M. Can we use statins to prevent stroke in Fabry disease?. J Inherit Metab Dis 32, 481–487 (2009). https://doi.org/10.1007/s10545-009-1156-2
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DOI: https://doi.org/10.1007/s10545-009-1156-2