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Specialty: Genetics & Stem Cells
Therapy: Dialysis
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Risk factors for severe clinical events in male and female patients with Fabry disease treated with agalsidase beta enzyme replacement therapy: Data from the Fabry Registry
Conclusions Patients with on-ERT severe events had more advanced Fabry organ involvement at baseline than those without such events and patients who initiated ERT at a younger age had less residual risk of on-ERT events. The observed patterns of residual risk may aid clinicians in multidisciplinary monitoring of male and female patients with Fabry disease receiving ERT, and in determining the need for administration of adjunctive therapies.
Source: Molecular Genetics and Metabolism - June 13, 2016 Category: Genetics & Stem Cells Source Type: research
