Fabry disease: A pharmacological chaperone on the horizon.
作者： Martina Gaggl , Gere Sunder-Plassmann
关键词: Pharmacological chaperone 、 In patient 、 Treatment options 、 Fabry disease 、 Medicine 、 Left ventricular mass 、 Enzyme replacement therapy 、 Pharmacology
摘要: For more than a decade, enzyme replacement therapy represented the only treatment option for patients with Fabry disease. New findings suggest that pharmacological chaperone can induce renal substrate clearance, decrease left ventricular mass and improve gastrointestinal symptoms in specific mutations GLA.
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