发明名称 |
Use of tetrahydrobiopterin as a marker and a therapeutic agent for Fabry disease |
摘要 |
<p>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.</p> |
申请公布号 |
AU2010233147(A1) |
申请公布日期 |
2011.10.13 |
申请号 |
AU20100233147 |
申请日期 |
2010.04.09 |
申请人 |
BAYLOR RESEARCH INSTITUTE |
发明人 |
SCHIFFMANN, RAPHAEL;BOTTIGLIERI, TEODORO G.;ARNING, ERLAND;MOORE, DAVID F. |
分类号 |
A61K31/195;A61K31/4985;A61K31/50;A61P3/00;A61P9/00;A61P43/00 |
主分类号 |
A61K31/195 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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