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
WO2010118330(A3)
申请公布日期
2011.03.31
申请号
WO2010US30552
申请日期
2010.04.09
申请人
BAYLOR RESEARCH INSTITUTE;SCHIFFMANN, RAPHAEL;BOTTIGLIERI, TEODORO, G.;ARNING, ERLAND;MOORE, DAVID, F.
发明人
SCHIFFMANN, RAPHAEL;BOTTIGLIERI, TEODORO, G.;ARNING, ERLAND;MOORE, DAVID, F.