Enzyme replacement therapy in two patients with classic Fabry disease from the same family tree: Two case reports.

Background: The pathophysiology of Fabry disease (FD)-induced progressive vital organ damage is irreversible. Disease progression can be delayed using enzyme replacement therapy (ERT). In patients with classic FD, sporadic accumulation of globotriaosylceramide (GL-3) in the heart and kidney begins ; however, until childhood, GL-3 accumulation is mild and reversible and can be restored by ERT.