Urinary triaosylceramide (Gb3) as a marker of cardiac disease
View Patent ↗The present invention describes a method for determination of urinary globotriaosylceramide (Gb3) levels in non-Fabry disease cardiac patients. Determination of Gb3 levels provides a screening method for determining cardiac risk and may offer an alternative therapeutic option for cardiac disease management or cardiac disease risk mitigation by lowering Gb3 levels by the use of pharmacological chaperones or other agents.
1. A method of treating a cardiac disease or condition in a non-Fabry disease subject comprising administering to the subject in need thereof an effective amount of 1-deoxygalactonojirimycin or a salt thereof, wherein the subject does not have Fabry disease.
2. The method of claim 1 , wherein the 1-deoxygalactonojirimycin or a salt thereof is administered in an amount to yield a Gb3 concentration in the urine of the subject of 200 ng/ml of lower.
3. The method of claim 1 , wherein 1-deoxygalactonojirimycin hydrochloride is administered.
4. The method of claim 1 , wherein the subject has no genetic predisposition towards Fabry disease.
5. The method of claim 1 , wherein the subject is a non-Fabry disease cardiac patient.
6. The method of claim 1 , wherein the cardiac disease or condition is selected form hypertrophic cardiomyopathy, cardiac rhythm and conduction defects, coronary artery disease, arrhythmia, conduction blocks and valvular disease.
7. The method of claim 3 , wherein the subject has no genetic predisposition towards Fabry disease.