A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/445 (2006.01) A61K 31/7088 (2006.01) A61K 35/28 (2006.01) A61K 38/47 (2006.01) A61K 48/00 (2006.01) A61P 3/00 (2006.01)
Patent
CA 2368812
The present invention provides the use of an inhibitor of glycolipid synthesis, such as N-butyldeoxynojirimycin (NB-DNJ), N-butyldeoxygalactonojirimycin (NB-DGJ) or N-nonyldeoxynojirimycin (NN-DNJ), and an agent capable of increasing the rate of glycolipid degradation in the manufacture of a medicament for the treatment of a disorder which has at least a component based on glycolipid storage. Such disorders include Gaucher disease, Sandhoff's disease, Fabry's disease. Tay-Sach's disease, Niemann-Pick C storage disease, GM1 gangliosidosis, genetic disorders in which neuronal glycolipid accumulation contributes to the disease's pathology, e.g. mucopolysaccharidoses, neurological disorders in which glucosylceramide- containing glycolipid accumulation contributes to the disease's pathology such as Alzheimer's disease, stroke and epilepsy, cancers of neuronal origin such as glioblastoma and astrocytoma and cancers originating outside neuronal tissue but presenting with neuronal metastases.
La présente invention concerne l'utilisation d'un inhibiteur de synthèse de glycolipide tel que le N-butyldéoxynojirimycine (NB-DNJ), le N-butyldéoxygalactonojirimycine (NB-DGJ) ou le N-nonyldéoxynojirimycine (NN-DNJ), et un agent capable d'accroître la vitesse de dégradation des glycolipides dans le processus de fabrication d'un médicament destiné au traitement d'un trouble pour lequel au moins un constituant est basé sur le stockage des glycolipides. Ces troubles comprennent la maladie de Gaucher, la maladie de Sandhoff, la maladie de Fabry, la maladie de Tay-Sach, la maladie de Niemann-Pick de type C, la gangliosidose GM1, les troubles génétiques dans lesquels l'accumulation de glycolipides dans les neurones contribue à la pathologie de la maladie, par exemple les mucopolysaccharidoses, les troubles neurologiques dans lesquels l'accumulation de glycolipides contenant du glucosylcéramide contribue à la pathologie de la maladie telle que la maladie d'Alzheimer, l'accident cérébrovasculaire et l'épilepsie, les cancers d'origine neuronale tels que le glioblastome et l'astrocytome et les cancers se développant à l'extérieur du tissu neuronal mais présentant cependant des métastases neuronales.
Butters Terence D.
Dwek Raymond A.
Jeyakumar Mylvaganam
Platt Frances M.
Priestman David
Actelion Pharmaceuticals Ltd
Oxford Glycosciences (uk) Limited
Smart & Biggar
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