Rare diseases Xagena
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 ...
