Adult Fabry patients in Sweden – Baseline data and Outcome of Four-Year Enzyme Replacement Treatment in a Male Cohort without Proteinuria
JMED Research
Background: Fabry disease is a rare X-linked lysosomal storage disease caused by defective activity of the lysosomal enzyme α-galactosidase A. The objective was to study the baseline characteristics of Fabry patients in Sweden, Furthermore, to study the effect of 4 years of ERT (enzyme replacement…