Substrate reduction intervention by l-cycloserine in twitcher mice (globoid cell leukodystrophy) on a B6;CAST/Ei background

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

Timothy M. Cox, Robin Lachmann, C. E. Hollak et al. · The Lancet · 2000 · 744 citations

Medicine, Pharmacology, Pharmacotherapy +4

Prevention of Lysosomal Storage in Tay-Sachs Mice Treated with <i>N</i> -Butyldeoxynojirimycin

Frances M. Platt, Gabrielle R. Neises, Gabriele Reinkensmeier et al. · Science · 1997 · 342 citations

Life Sciences, Biochemistry, Neuroscience +14

Krabbe disease: a galactosylsphingosine (psychosine) lipidosis.

Lars Svennerholm, M. T. Vanier, J.‐E. Månsson · Journal of Lipid Research · 1980 · 323 citations · Full text

Life Sciences, Biochemistry, Neurology +14

Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with <i>N</i> -butyldeoxynojirimycin

Mylvaganam Jeyakumar, Terry D. Butters, Mario Cortina‐Borja et al. · Proceedings of the National Academy of Sciences · 1999 · 269 citations · Full text

Life Sciences, Neurology, Neuroscience +17

Enhanced survival in Sandhoff disease mice receiving a combination of substrate deprivation therapy and bone marrow transplantation

Mylvaganam Jeyakumar, Francine Norflus, Cynthia J. Tifft et al. · Blood · 2001 · 118 citations

Medicine, Immunology, Pathology +6