Use of tetrahydrobiopterin as a marker and a therapeutic...

Details Use of tetrahydrobiopterin as a marker and a therapeutic... Use of tetrahydrobiopterin as a marker and a therapeutic...

A - Human Necessities

61

K

A61K 31/50 (2006.01) A61K 31/195 (2006.01) A61K 31/4985 (2006.01) A61P 3/00 (2006.01) A61P 9/00 (2006.01) A61P 43/00 (2006.01)

Patent

CA 2756272

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.

La présente invention concerne des marqueurs sanguins et tissulaires destinés à déterminer l'état métabolique, le risque de complications de l'état de santé chez les patients atteints de la maladie de Fabry, lesdits marqueurs étant utilisés en tant que biomarqueurs permettant de suivre l'efficacité du traitement dans des modèles animaux et chez des patients atteints de la maladie de Fabry comprenant la tétrahydrobioptérine (BH4), des précurseurs et des métabolites de BH4, et autres co-facteurs associés. La présente invention concerne en outre l'utilisation du traitement par BH4 comme option thérapeutique dans la maladie de Fabry pour prévenir, ralentir ou inverser les manifestations cardiovasculaires et rénales de la maladie de Fabry.

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