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Renal function predicts long-term outcome on enzyme replacement therapy in patients with Fabry disease

Nephrology Dialysis Transplantation Dec 16, 2017

Lenders M, et al. - In Fabry disease (FD) patients receiving long-term enzyme replacement therapy (ERT), the incidence of FD-related renal, cardiac and neurologic end points were assessed. In these patients, FD progression on ERT was determined by even moderately impaired renal function, over and above age. Findings call for renal and cardiac protection to prevent patients from deleterious manifestations of the disease.
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