Fabry disease: A pharmacological chaperone on the horizon

Nature Reviews Nephrology 12, 653 (2016). doi:10.1038/nrneph.2016.138 Authors: Martina Gaggl & Gere Sunder-Plassmann For more than a decade, enzyme replacement therapy represented the only treatment option for patients with Fabry disease. New findings suggest that a pharmacological chaperone can induce renal substrate clearance, decrease left ventricular mass and improve gastrointestinal symptoms in patients with specific mutations in GLA.
Source: Nature Reviews Nephrology - Category: Urology & Nephrology Authors: Tags: News and Views Source Type: research