COMBINATION OF N-BUTYLDEOXYNOJIRIMYCIN (NB-DNJ) AND GLYCOLIPID DEGRADING ENZYMES IN THERAPY

The present invention relates to the use of a glucosylceramide synthesis inhibitor, such as N-butyldeoxygalactonojirimycin (NB-DGJ), in the manufacture of a medicament for use in combination therapy in the treatment of disorders which have at least a component based on glycolipid storage. Such disea...

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Hauptverfasser: DWEK, RAYMOND, A, JEYAKUMAR, MYLVAGANAM, PRIESTMAN, DAVID, PLATT, FRANCES, M, BUTTERS, TERENCE, D
Format: Patent
Sprache:eng ; fre ; ger
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Zusammenfassung:The present invention relates to the use of a glucosylceramide synthesis inhibitor, such as N-butyldeoxygalactonojirimycin (NB-DGJ), in the manufacture of a medicament for use in combination therapy in the treatment of disorders which have at least a component based on glycolipid storage. Such diseases include Niemann-Pick C storage disease, Gaucher disease, Sandhoff disease, Tay-Sachs disease, GM1 gangliosidosis, Fabrys disease, Alzheimer's disease, stroke, epilepsy and cancers such as glioblastoma and astrocytoma.