Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis
Top Cited Papers
- 1 April 2000
- journal article
- clinical trial
- Published by Elsevier in The Lancet
- Vol. 355 (9214), 1481-1485
- https://doi.org/10.1016/s0140-6736(00)02161-9
Abstract
No abstract availableKeywords
This publication has 18 references indexed in Scilit:
- N-butyldeoxygalactonojirimycin: a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivoBiochemical Pharmacology, 2000
- Delayed symptom onset and increased life expectancy in Sandhoff disease mice treated with N -butyldeoxynojirimycinProceedings of the National Academy of Sciences, 1999
- Mice with disrupted GM2/GD2 synthase gene lack complex gangliosides but exhibit only subtle defects in their nervous system.Proceedings of the National Academy of Sciences, 1996
- The Tolerability and Pharmacokinetics of N-Butyl-deoxynojirimycin in Patients with Advanced HIV Disease (ACTG 100)JAIDS Journal of Acquired Immune Deficiency Syndromes, 1995
- Replacement therapy with imiglucerase for type 1 Gaucher's diseaseThe Lancet, 1995
- Enzyme Therapy in Type 1 Gaucher Disease: Comparative Efficacy of Mannose-Terminated Glucocerebrosidase from Natural and Recombinant SourcesAnnals of Internal Medicine, 1995
- Marked elevation of plasma chitotriosidase activity. A novel hallmark of Gaucher disease.Journal of Clinical Investigation, 1994
- Gaucher DiseaseMedicine, 1992
- Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's DiseaseNew England Journal of Medicine, 1991
- Partial Enzyme Deficiencies: Residual Activities and the Development of Neurological DisordersDevelopmental Neuroscience, 1983