Claims
- 1. A method of treating Niemann-Pick type C disease by administration of a therapeutically effective amount of an inhibitor of glucosylceramide synthesis.
- 2. The method of claim 1, wherein the inhibitor of glucosylceramide synthesis is an imido sugar.
- 3. The method of claim 2, wherein the imido sugar is selected from the group consisting of N-butyldeoxynojirimycin, N-butyldeoxygalactonojirimycin, and N-nonyldeoxynojirimycin.
- 4. The method of claim 1, wherein the inhibitor is selected from the group consisting of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol or a structurally related analogue thereof.
- 5. The method of claim 1, wherein the inhibitor is a nucleic acid encoding a peptide or protein inhibitor of glucosylceramide synthesis, or an antisense sequence or catalytic RNA capable of interfering with the expression of enzymes responsible for glucosylceramide synthesis.
- 6. The method of claim 1, wherein the inhibitor of glucosylceramide synthesis is an inhibitor of glucosylceramide synthase.
- 7. A pharmaceutical composition comprising an inhibitor of glucosylceramide synthesis selected from the group of N-butyldeoxynojirimycin, N-butyldeoxygalactonojirimycin, N-nonyldeoxynojirimycin, 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol, and structurally related analogues thereof, and a pharmaceutically acceptable carrier.
- 8. A method of treating Niemann-Pick type C disease by administration of a therapeutically effective amount of an agent capable of increasing the rate of neuronal glycolipid degradation.
- 9. The method of claim 8, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a neuronal glycolipid degrading enzyme.
- 10. The method of claim 9, wherein the neuronal glycolipid degrading enzyme is a lysosomal hexoseaminidase, a galactosidase, a sialidase and glucosylceramide glucosidase.
- 11. The method of claim 8, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a molecule which increases the activity of a glycolipid degrading enzyme.
- 12. The method of claim 8, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a nucleic acid sequence which encodes a neuronal glycolipid degrading enzyme.
- 13. A method of treating Alzheimer's disease by administration of a therapeutically effective amount of an inhibitor of glucosylceramide synthesis.
- 14. The method of claim 13, wherein the inhibitor of glucosylceramide synthesis is an imido sugar.
- 15. The method of claim 14, wherein the imido sugar is selected from the group consisting of N-butyldeoxynojirimycin, N-butyldeoxygalactonojirimycin, and N-nonyldeoxynojirimycin.
- 16. The method of claim 13, wherein the inhibitor is selected from the group consisting of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol or a structurally related analogue thereof.
- 17. The method of claim 13, wherein the inhibitor is a nucleic acid encoding a peptide or protein inhibitor of glucosylceramide synthesis, or an antisense sequence or catalytic RNA capable of interfering with the expression of enzymes responsible for glucosylceramide synthesis.
- 18. The method of claim 13, wherein the inhibitor of glucosylceramide synthesis is an inhibitor of glucosylceramide synthase.
- 19. A method of treating Alzheimer's disease by administration of a therapeutically effective amount of an agent capable of increasing the rate of neuronal glycolipid degradation.
- 20. The method of claim 19, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a lysosomal hexoseaminidase, a galactosidase, a sialidase, and/or a glucosylceramide glucosidase.
- 21. The method of claim 19, wherein the agent is a molecule capable of increasing the activity of a glycolipid degrading enzyme.
- 22. A method of treating Alzheimer's disease by administration of a therapeutically effective amount of an agent capable of increasing the rate of neuronal glycolipid degradation.
- 23. The method of claim 22, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a neuronal glycolipid degrading enzyme.
- 24. The method of claim 23, wherein the neuronal glycolipid degrading enzyme is a lysosomal hexoseaminidase, a galactosidase, a sialidase and glucosylceramide glucosidase.
- 25. The method of claim 22, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a molecule which increases the activity of a glycolipid degrading enzyme.
- 26. The method of claim 22, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a nucleic acid sequence which encodes a neuronal glycolipid degrading enzyme.
- 27. A method of treating or ameliorating epilepsy, comprising administering a therapeutically effective amount of an inhibitor of glucosylceramide synthesis.
- 28. The method of claim 27, wherein the inhibitor of glucosylceramide synthesis is an imido sugar.
- 29. The method of claim 28, wherein the imido sugar is selected from the group consisting of N-butyldeoxynojirimycin, N-butyldeoxygalactonojirimycin, and N-nonyldeoxynojirimycin.
- 30. The method of claim 27, wherein the inhibitor is selected from the group consisting of 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol or a structurally related analogue thereof.
- 31. The method of claim 27, wherein the inhibitor is a nucleic acid encoding a peptide or protein inhibitor of glucosylceramide synthesis, or an antisense sequence or catalytic RNA capable of interfering with the expression of enzymes responsible for glucosylceramide synthesis.
- 32. The method of claim 27, wherein the inhibitor of glucosylceramide synthesis is an inhibitor of glucosylceramide synthase.
- 33. A method of treating or ameliorating epilepsy, comprising administering a therapeutically effective amount of an agent capable of increasing the rate of neuronal glycolipid degradation.
- 34. The method of claim 33, wherein the agent capable of increasing the rate of neuronal glycolipid degradation is a lysosomal hexoseaminidase, a galactosidase, a sialidase, and/or a glucosylceramide glucosidase.
- 35. The method of claim 33, wherein the agent is a molecule capable of increasing the activity of a glycolipid degrading enzyme.
- 36. A method of treating or ameliorating a ganglioside-related disorder, comprising administering a therapeutically effective amount of a ganglioside.
- 37. The method of claim 36, wherein the ganglioside is GM1 ganglioside.
- 38. The method of claim 36, wherein the ganglioside-related disorder is selected from the group consisting of Parkinson's disease, stroke, and spinal cord injuries.
- 39. A pharmaceutical composition useful for treatment of Parkinson's disease, stroke, and spinal cord injuries, comprising a ganglioside and a pharmaceutically acceptable carrier.
- 40. The pharmaceutical composition of claim 39, wherein the ganglioside is GM1 ganglioside.
- 41. The pharmaceutical composition of claim 39, further comprising an inhibitor of glucosylceramide synthesis.
- 42. The pharmaceutical composition of claim 41, wherein the glucosylceramide synthesis inhibitor is an imino sugar.
- 43. The pharmaceutical composition of claim 42, wherein the imino sugar is selected from the group consisting of N-butyldeoxynojirimycin, N-butyldeoxygalactonojirimycin, and N-nonyldeoxynojirimycin.
- 44. The pharmaceutical composition of claim 41, wherein the inhibitor of glucosylceramide synthesis is 1-phenyl-2-decanoylamino-3-morpholino-1-propanol (PDMP), D-threo-1-phenyl-2-decanoylamino-3-morpholino-1-propanol and structurally related analogues thereof.
- 45. The pharmaceutical composition of claim 39, further comprising a therapeutically effective amount of an agent capable of increasing the rate of neuronal glycolipid degradation.
- 46. The pharmaceutical composition of claim 45, wherein the agent is a neuronal glycolipid degrading enzyme.
- 47. The pharmaceutical composition of claim 46, wherein the neuronal glycolipid degrading enzyme is selected from the group comprising a lysosomal hexoseaminidase, a galactosidase, a sialidase and glucosylceramide glucosidase.
- 48. The pharmaceutical composition of claim 45, wherein the agent is a molecule which increases the activity of a glycolipid degrading enzyme.
- 49. The pharmaceutical composition of claim 45, wherein the agent is a nucleic acid sequence which encodes a neuronal glycolipid degrading enzyme.
Priority Claims (1)
Number |
Date |
Country |
Kind |
9909064.9 |
Apr 2000 |
GB |
|
RELATED PATENT APPLICATIONS
[0001] This application is a continuation of PCT/GB00/01563 filed Apr. 20, 2000, which application is herein specifically incorporated by reference.
Continuations (1)
|
Number |
Date |
Country |
Parent |
PCT/GB00/01563 |
Apr 2000 |
US |
Child |
10007306 |
Oct 2001 |
US |