Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis.

BACKGROUND: Current treatment for Gaucher's disease involves administration of intravenous glucocerebrosidase to degrade glucocerebroside stored in lysosomes. Lowering the rate of biosynthesis of glucocerebroside should decrease accumulation of this substrate. We investigated the safety and eff...

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Main Authors: Cox, T, Lachmann, R, Hollak, C, Aerts, J, van Weely, S, Hrebícek, M, Platt, F, Butters, T, Dwek, R, Moyses, C, Gow, I, Elstein, D, Zimran, A
格式: Journal article
語言:English
出版: 2000