Substrate reduction therapy
AssigneesThe United States of America, as represented by the Secretary, Department of Health and Human Services, The Chancellor, Masters and Scholars of the University of Oxford
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.
Claims3
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.
Dependencies are inferred from the claim text.