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Search results for "Fabry disease"

Fabry disease is a rare X-linked hereditary disease caused by mutations in the GLA gene encoding the lysosomal enzyme alpha-galactosidase. Males with classical disease are severely affected, while in ...


Enzyme replacement therapy ( ERT ) is the standard therapy for Fabry disease. Enzyme replacement therapy partially cleared microvascular deposits of GL-3 from the heart, kidney and skin of most Fabry ...