The study evaluated the safety and efficacy of recombinant human α-galactosidase A (rhα-GalA) replacement therapy in patients with Fabry's disease, a lysosomal storage disorder characterized by the accumulation of globotriaosylceramide and related glycosphingolipids. The multicenter, randomized, placebo-controlled, double-blind trial involved 58 patients who received rhα-GalA every 2 weeks for 20 weeks, followed by an open-label extension study. The primary endpoint was the percentage of patients with clearance of renal microvascular endothelial deposits of globotriaosylceramide. Patients in the rhα-GalA group had significantly higher rates of clearance compared to the placebo group (69% vs. 0%, P<0.001). The study also evaluated histologic clearance in the endomyocardium and skin, changes in pain levels, and quality of life. After six months of open-label therapy, all patients in the former placebo group and 98% of those in the former rhα-GalA group had clearance of microvascular endothelial deposits. Mild-to-moderate infusion reactions were more common in the rhα-GalA group. The results suggest that rhα-GalA replacement therapy effectively clears microvascular endothelial deposits of globotriaosylceramide, reversing the pathogenesis of key clinical manifestations of Fabry's disease.The study evaluated the safety and efficacy of recombinant human α-galactosidase A (rhα-GalA) replacement therapy in patients with Fabry's disease, a lysosomal storage disorder characterized by the accumulation of globotriaosylceramide and related glycosphingolipids. The multicenter, randomized, placebo-controlled, double-blind trial involved 58 patients who received rhα-GalA every 2 weeks for 20 weeks, followed by an open-label extension study. The primary endpoint was the percentage of patients with clearance of renal microvascular endothelial deposits of globotriaosylceramide. Patients in the rhα-GalA group had significantly higher rates of clearance compared to the placebo group (69% vs. 0%, P<0.001). The study also evaluated histologic clearance in the endomyocardium and skin, changes in pain levels, and quality of life. After six months of open-label therapy, all patients in the former placebo group and 98% of those in the former rhα-GalA group had clearance of microvascular endothelial deposits. Mild-to-moderate infusion reactions were more common in the rhα-GalA group. The results suggest that rhα-GalA replacement therapy effectively clears microvascular endothelial deposits of globotriaosylceramide, reversing the pathogenesis of key clinical manifestations of Fabry's disease.