Jakubowska Ewa, Ryba Monika, Hruby Zbigniew
Oddział Nefrologiczny z Pododdziałem Diabetologicznym i Transplantacyjnym, Wrocław.
Przegl Lek. 2006;63(4):218-9.
Fabry disease is a rare genetic disorder, which is linked to a defect of alfa-galactosidase. In consequence it leads to an excess of glicosphyngolipids in lysosomes of various tissues and organs. Clinical symptoms are related to heart, skin, kidneys and nervous system. Nowadays due to a possibility of substitution of galactosidase A, a influence on clinical course of the disease can be attained: arresting of progression and avoidance of complications.
法布里病是一种罕见的遗传性疾病,与α-半乳糖苷酶缺陷有关。因此,它会导致各种组织和器官的溶酶体中糖鞘脂过量。临床症状与心脏、皮肤、肾脏和神经系统有关。如今,由于有可能替代半乳糖苷酶A,因此可以对疾病的临床进程产生影响:阻止病情进展并避免并发症。