Method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
US-9181184-B2 · Nov 10, 2015 · US
Method to predict response to pharmacological chaperone treatment of diseases
US9545397B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9545397-B2 |
| Application number | US-201514731603-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jun 5, 2015 |
| Priority date | Feb 12, 2008 |
| Publication date | Jan 17, 2017 |
| Grant date | Jan 17, 2017 |
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- Title
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Abstract
Official abstract text for this publication.
The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
First claim
Opening claim text (preview).
What is claimed is: 1. A method of treating a patient diagnosed with Pompe disease which comprises administering to the patient a therapeutically effective dose of 1-deoxynojirimycin, wherein the patient expresses a mutant a-glucosidase selected from the group consisting of the a-glucosidase mutations E262K, P266S, P285R, P285S, L291F, L291H, L291P, M318K, G377R, A445P, Y455C, Y455F, P457L, G483R, G483V, M519V, S529V, P545L, G549R, L552P, Y575S, E579K, A610V, H612Q, A644P, and AN470.
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Antihyperlipidemics · CPC title
for hyperglycaemia, e.g. antidiabetics · CPC title
Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title
Drugs for disorders of the metabolism (of the blood or the extracellular fluid A61P7/00) · CPC title
involving hydrolase · CPC title
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- What does patent US9545397B2 cover?
- The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining α-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of α-galactosid…
- Who is the assignee on this patent?
- Amicus Therapeutics Inc
- What technology area does this patent fall under?
- Primary CPC classification A61K31/445. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Tue Jan 17 2017 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 1 related publication on this page (citations in our corpus or others sharing the same primary CPC).