Substrate reduction therapy
AssigneesThe Chancellor, Masters and Scholars of the University of Oxford, The United States of America, as represented by the Secretary, Department of Health and Human Services
InventorsFrances Mary Platt, Emyr Lloyd-Evans, Forbes Dennison Porter
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Abstract
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.
Claims13
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 Smith-Lemli-Opitz Syndrome (SLOS), CHILD syndrome, XLD chondrodysplasia punctata type 2, Conradi-Hünnermann-Happle syndrome, or HEM dysplasia, the method comprising selecting the subject having the disease, and administering to the subject an effective amount of an inhibitor of sphingolipid biosynthesis wherein the inhibitor of sphingolipid biosynthesis is N-butyldeoxynojirimycin;
- N-nonyldeoxynojirimycin;
- N-butyldeoxygalactonojirimycin;
- N-5-adamantane-1-yl-methoxypentyl-deoxynojirimycin;
- alpha-homogalactonojirimycin;
- nojirimycin;
- deoxynojirimycin;
- N7-oxadecyl-deoxynojirimycin;
- deoxygalactonojirimycin;
- N-butyl-deoxygalactonojirimycin;
- N-nonyl-deoxygalactonojirimycin;
- N-nonyl-6deoxygalactonojirimycin;
- N7-oxanonyl-6deoxy-DGJ;
- alpha-homoallonojirimycin;
- or beta-1-C-butyl-deoxygalactonojirimycin.
Dependencies are inferred from the claim text.