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: Martina Gaggl Gere Sunder-Plassmann Tags: News and Views Source Type: research
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