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Substrate reduction therapy
US9428541B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9428541-B2 |
| Application number | US-201314026957-A |
| Country | US |
| Kind code | B2 |
| Filing date | Sep 13, 2013 |
| Priority date | Jun 27, 2007 |
| Publication date | Aug 30, 2016 |
| Grant date | Aug 30, 2016 |
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- Title
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Abstract
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The present invention provides a compound which is an inhibitor of sphingolipid biosynthesis for use in the treatment of a disease which has a secondary Niemann-Pick type C disease like cellular phenotype.
First claim
Opening claim text (preview).
The invention claimed is: 1. A method of treating a subject having a disease which has a secondary Niemann-Pick type C disease like cellular phenotype, wherein the disease is inborn error of cholesterol synthesis, Tangier disease, Pelizaeus-Merzbacher disease, Mucolipidosis II (cell), or variant late infantile-Neuronal Ceroid Lipofuscinosis, and wherein the method comprises selecting the subject having the disease, and administering to the subject an effective amount of N-butyldeoxynojirimycin; N-nonyldeoxynojirimycin; N-butyldeoxygalactonojirimycin; N-5adamantane-1-yl-methoxypentyl-deoxynojirimycin; alpha-homogalactonojirimycin; nojirimycin; deoxynojirimycin; N7-oxadecyl-deoxynojirimycin; deoxygalactonojirimycin; N-butyl-deoxygalactonojirimycin; N-nonyl-6deoxy-DGJ; alpha-homoallonojirimycin; or beta-1C-butyl-deoxygalactonojirimycin. 2. The method of claim 1 , wherein the disease is Tangier disease. 3. The method of claim 2 , wherein the inhibitor of sphingolipid biosynthesis is N-butyldeoxynojirimycin.
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Classifications
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
for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia · CPC title
Drugs for dermatological disorders · CPC title
by only one oxygen atom doubly bound · CPC title
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- What does patent US9428541B2 cover?
- The present invention provides a compound which is an inhibitor of sphingolipid biosynthesis for use in the treatment of a disease which has a secondary Niemann-Pick type C disease like cellular phenotype.
- Who is the assignee on this patent?
- Us Health, The Chancellor Masters And Scholars Of The Univ Of Oxford, The Chancellor Masters And Scholars Of The Univ Of Oxford
- 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 Aug 30 2016 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 8 related publications on this page (citations in our corpus or others sharing the same primary CPC).