10 Year Outcome of Enzyme Replacement Therapy with Agalsidase Beta in Patients with Fabry Disease.
The aim of this study was to assess the clinical effect of α-galactosidase. Following supplementation, participants were assessed for severe clinical events, renal function and cardiac structure. This 10-year study documents the effectiveness of agalsidase beta (1 mg/kg/2 weeks) in patients with Fabry disease. Most patients remained alive and event-free. Patients who initiated treatment at a younger …