TreatmentforFabrydisease

Fabrydisease–isararehereditary disease involving kidneys, heart, central and peripheral nervous system, skin, eyes and other organs. Enzyme replacement therapy with recombinant agalsidase alfa and beta for Fabry disease is available since 2001. Numerous studies, including double blind, placebo-controlled clinical trials, showed that enzyme replacement therapy retards progression of kidney dysfunction, left ventricular hypertrophy and central nervous system disease, lessens neuropathic pain, improves health-related quality of life and decreases a risk of unfavorable outcomes in patients with Fabry diseases.

Key words

Fabry disease, treatment, outcomes.