Use of tetrahydrobiopterin as a marker and a therapeutic agent for fabry disease
AssigneeBaylor Research Institute
InventorsRaphael Schiffmann, Teodoro G. Bottiglieri, Erland Arning, David F. Moore
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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.
Claims5
A method of treating Fabry's disease in one or more subjects comprising the steps of:
- identifying the one or more subjects in need of treatment against Fabry's disease;
- and
- administering one or more pharmaceutical compositions comprising a therapeutically effective amount of tetrahydrobiopterin (BH4) in a therapeutically effective amount sufficient to treat Fabry's disease.
Dependencies are inferred from the claim text.