The role of the iminosugar N‐butyldeoxynojirimycin (miglustat) in the management of type I (non‐neuronopathic) Gaucher disease: A position statement
- 1 September 2003
- journal article
- review article
- Published by Wiley in Journal of Inherited Metabolic Disease
- Vol. 26 (6) , 513-526
- https://doi.org/10.1023/a:1025902113005
Abstract
N-Butyldeoxynojirimycin (NB-DNJ, miglustat 'Zavesca') is an orallyactive iminosugar which inhibits the biosynthesis of macromolecular substrates that accumulate pathologically in...Keywords
This publication has 48 references indexed in Scilit:
- Low-Dose N-Butyldeoxynojirimycin (OGT 918) for Type I Gaucher DiseaseBlood Cells, Molecules, and Diseases, 2002
- High prevalence of low serum vitamin B12 in a multi‐ethnic Israeli populationBritish Journal of Haematology, 2001
- Dixon Quantitative Chemical Shift Imaging Is a Sensitive Tool for the Evaluation of Bone Marrow Responses to Individualized Doses of Enzyme Supplementation Therapy in Type 1 Gaucher DiseaseBlood Cells, Molecules, and Diseases, 2001
- Mutation analysis of the acid β-glucosidase gene in a patient with type 3 Gaucher disease and neutralizing antibody to algluceraseMutation Research - Fundamental and Molecular Mechanisms of Mutagenesis, 2001
- Substrate reduction therapy for glycosphingolipid storage disordersExpert Opinion on Investigational Drugs, 2001
- Effect of Low-dose Enzyme Replacement Therapy on Bones in Gaucher Disease Patients with Severe Skeletal InvolvementBlood Cells, Molecules, and Diseases, 1996
- The Clinical Course of Treated and Untreated Gaucher Disease. A Study of 45 PatientsBlood Cells, Molecules, and Diseases, 1995
- 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