Invention Grant
US08410101B2 Use of tetrahydrobiopterin as a marker and a therapeutic agent for fabry disease 有权
使用四氢生物喋呤作为标记物和药物治疗剂

Use of tetrahydrobiopterin as a marker and a therapeutic agent for fabry disease
Abstract:
Blood and tissue markers of the metabolic status, risk of health complications in Fabry disease patients and as a biomarker to follow the efficacy of treatment in animal models and patients with Fabry disease comprising tetrahydrobiopterin (BH4), precursors and metabolites of BH4, and other related co-factors is disclosed herein. The present invention further describes the use of BH4 therapy as a treatment option for Fabry disease to prevent, slow or reverses vascular cardiac and renal manifestations of Fabry disease.
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