Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease. por Elstein, D, Hollak, C, Aerts, J, van Weely, S, Maas, M, Cox, T, Lachmann, R, Hrebicek, M, Platt, F, Butters, T, Dwek, R, Zimran, A

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

Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy. por Platt, F, Jeyakumar, M, Andersson, U, Priestman, D, Dwek, R, Butters, T, Cox, T, Lachmann, R, Hollak, C, Aerts, J, Van Weely, S, Hrebícek, M, Moyses, C, Gow, I, Elstein, D, Zimran, A