IP Library › Granted Patent US 8,404,668
Granted Patent B2
US 8,404,668 · App. 13/248,434 · Granted Mar 26, 2013

Method of treating gaucher disease

Inventors: Don J. Mahuran (Toronto, CA); Michael B. Tropak (Toronto, CA); Justin D. Buttner (Rosehill, AU); Jan E. Blanchard (St. Catharines, CA); Eric D. Brown (Oakville, CA)
Assignees: The Hospital for Sick Children; McMaster University
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 8,404,668
App. No.
13/248,434
Granted
Mar 26, 2013
Kind
B2
Abstract

Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.

Claims (5)

1. A composition comprising (i) ambroxol or a pharmaceutically acceptable salt thereof; and (ii) a glucocerebrosidase.

2. The composition according to claim 1 comprising ambroxol hydrochloride.

3. The composition according to claim 1 , wherein the glucocerebrosidase is a wild type glucocerebrosidase.

4. The composition according to claim 1 , wherein the glucocerebrosidase is beta-glucocerebrosidase.

5. The composition according to claim 2 , wherein the glucocerebrosidase is beta-glucocerebrosidase.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 15, 2012
From: MAHURAN, DON J.; TROPAK, MICHAEL B.; BUTTNER, JUSTIN D.
To: THE HOSPITAL FOR SICK CHILDREN
Reel/Frame 029301/0930 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Nov 15, 2012
From: BLANCHARD, JAN E.; BROWN, ERIC D.
To: MCMASTER UNIVERSITY
Reel/Frame 029302/0001 →
Continuity (5)
Division 12229445 · Aug 22, 2008
Provisional Application 60972968 · Sep 17, 2007
Provisional Application 61065550 · Feb 12, 2008
Provisional Application 61065684 · Feb 13, 2008
Related Publication 20120021487A1 · Jan 26, 2012