IP Library Granted Patent US 9,750,732
Granted Patent B2
US 9,750,732 · App. 14/729,630 · Granted Sep 5, 2017

Urinary triaosylceramide (Gb3) as a marker of cardiac disease

Inventors: Raphael Schiffmann (Rockwall, TX); Fanny Mochel (Dallas, TX); Lawrence Sweetman (Heath, TX); Sabrina Forni (Dallas, TX)
A61K31/445A61B10/007A61B10/0051A61B10/0058A61B2010/0061G01N2030/8831
View Patent ↗
Loading inventors, assignments & file history…
Monitor This Case
Get email alerts when status or documents change.
Order Certified Copies
Most orders are placed with the USPTO same day — all within 24 business hours.
Order via The Patent Place →
Pre-filled with this patent's details
Quick Facts
Patent No.
US 9,750,732
App. No.
14/729,630
Granted
Sep 5, 2017
Kind
B2
Abstract

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.

Claims (7)

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.

Assignments (3)
RELEASE OF SECURITY INTEREST Recorded Apr 27, 2026
From: WILMINGTON TRUST, NATIONAL ASSOCIATION
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 075494/0030 →
SECURITY INTEREST Recorded Apr 27, 2026
From: BIOMARIN PHARMACEUTICAL INC.; AMICUS THERAPEUTICS, INC.
To: CITIBANK, N.A., AS COLLATERAL AGENT
Reel/Frame 075493/0968 →
SECURITY INTEREST Recorded Oct 6, 2023
From: AMICUS THERAPEUTICS, INC.
To: WILMINGTON TRUST, NATIONAL ASSOCIATION
Reel/Frame 065177/0196 →
Continuity (3)
Division 12948693 · Nov 17, 2010
Provisional Application 61261831 · Nov 17, 2009
Related Publication 20150342939A1 · Dec 3, 2015