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

By T. Cox, R. Lachmann, C. Hollak, J. Aerts, S. van Weely, M. Hřebíček, F. Platt, T. Butters, R. Dwek, C. Moyses and 3 more. The Lancet, 2000

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Glycosidase inhibitors: update and perspectives on practical use. (2003, background): “The result of this clinical trial indicates that NB-DNJ (OGT 918) is effective in depleting GSLs and leads to an improvement in clinical parameters measuring disease progression (Cox et al., 2000).”
Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males (2001, background): “Another potentially therapeutic approach is substrate deprivation by inhibitors of glycosphyngolipid synthesis.”
The cell biology of lysosomal storage disorders (2004, positive): “The latter has been attempted for the sphingolipidoses, in which inhibition of glycolipid synthesis by N- butyl-deoxynojirimycin blocks the development of disease symptoms in Tay–Sachs-disease mouse models 94 and in Gaucher-disease patients 95,96 .”