Politei J M
Neurology Service, Juan A Fernandez Hospital, Buenos Aires, Argentina.
J Inherit Metab Dis. 2009 Aug;32(4):481-7. doi: 10.1007/s10545-009-1156-2. Epub 2009 Jun 2.
Fabry disease is a rare, X-linked lysosomal storage disease caused by an inborn deficiency of alpha-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.
To evaluate evidence and potential beneficial effects of statins in the vasculopathy of Fabry disease.
法布里病是一种罕见的X连锁溶酶体贮积病，由α-半乳糖苷酶A先天性缺乏引起，导致球三糖神经酰胺和其他中性糖脂在一系列细胞和组织中进行性蓄积。与肾和心脏功能不全相关，脑血管并发症可导致患者死亡。已经描述了几种导致血管损伤并进而发展为深部白质病变的机制。最近的临床试验强烈表明，他汀类药物通过神经保护特性或多效性作用预防中风。
评估他汀类药物在法布里病血管病变中的证据及潜在有益作用。
