IP Library Granted Patent US 9,480,682
Granted Patent B2
US 9,480,682 · App. 14/641,707 · Granted Nov 1, 2016

Methods for treatment of Fabry disease

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Quick Facts
Patent No.
US 9,480,682
App. No.
14/641,707
Granted
Nov 1, 2016
Kind
B2
Abstract

Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.

Claims (14)

1. A method for treatment of Fabry disease in a patient in need thereof, the method comprising administering to the patient a formulation comprising 150 mg of 1-deoxygalactonojirimycin or a salt thereof every other day.

2. The method of claim 1 , wherein the formulation is administered for at least 2 weeks.

3. The method of claim 1 , wherein the salt of 1-deoxygalactonojirimycin comprises migalastat hydrochloride.

4. The method of claim 1 , wherein the formulation comprises an oral dosage form.

5. The method of claim 4 , wherein the oral dosage form comprises a tablet, a capsule or a solution.

6. The method of claim 1 , wherein the formulation comprises an injectable solution.

7. The method of claim 6 , wherein the injectable solution comprises a carrier comprising water, an aqueous saline solution, an aqueous dextrose solution or an aqueous glycerol solution.

8. The method of claim 7 , wherein the carrier comprises a sterile liquid.

9. The method of claim 1 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity.

10. A method for treatment of Fabry disease in a patient in need thereof, the method comprising administering to the patient a formulation comprising 1-deoxygalactonojirimycin or a salt thereof, wherein the 1-deoxygalactonojirimycin or salt thereof is administered to the patient in an amount of 150 mg every other day for at least 2 weeks.

11. The method of claim 10 , wherein the 1-deoxygalactonojirimycin or salt thereof is administered orally.

12. The method of claim 10 , wherein the 1-deoxygalactonojirimycin or salt thereof is administered by injection.

13. The method of claim 10 , wherein the salt of 1-deoxygalactonojirimycin comprises migalastat hydrochloride.

14. The method of claim 10 , wherein the 1-deoxygalactonojirimycin or salt thereof enhances α-galactosidase A activity.

Assignments (10)
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 →
RELEASE OF SECURITY INTEREST Recorded Oct 6, 2023
From: HAYFIN SERVICES LLP
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 065164/0945 →
SECURITY INTEREST Recorded Jul 30, 2020
From: AMICUS THERAPEUTICS, INC.
To: HAYFIN SERVICES LLP, AS AGENT
Reel/Frame 053365/0342 →
RELEASE OF SECURITY INTEREST Recorded Jul 30, 2020
From: BPCR LIMITED PARTNERSHIP
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 053360/0659 →
OMNIBUS CONFIRMATION OF ASSIGNMENT AGREEMENT Recorded May 21, 2020
From: BIOPHARMA CREDIT PLC
To: BPCR LIMITED PARTNERSHIP
Reel/Frame 052741/0173 →
SECURITY INTEREST Recorded May 11, 2020
From: AMICUS THERAPEUTICS, INC.
To: BIOPHARMA CREDIT PLC
Reel/Frame 052625/0916 →
SECURITY INTEREST Recorded Sep 28, 2018
From: AMICUS THERAPEUTICS, INC.
To: BIOPHARMA CREDIT PLC
Reel/Frame 047004/0208 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Sep 6, 2018
From: LOCKHART, DAVID J.; CASTELLI, JEFF
To: AMICUS THERAPEUTICS, INC.
Reel/Frame 046799/0464 →