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| http://linked.open...gbank/description
| - Miglustat is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. Miglustat is marketed under the trade name Zavesca. Miglustat is now the first and only approved therapy for patients with Niemann-Pick disease type C (NP-C). It has recently been approved for treatment of progressive neurological symptoms in adult and pediatric patients in the European Union, Brazil, and South Korea. Miglustat was first developed as an anti-HIV agent in the 1990s. However, clinical experience with miglustat showed that therapeutic levels of the drug could not be achieved in patients without a high incidence of adverse effect. (en)
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| http://linked.open...y/drugbank/dosage
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| http://linked.open...generalReferences
| - # van Giersbergen PL, Dingemanse J: Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. J Clin Pharmacol. 2007 Oct;47(10):1277-82. Epub 2007 Aug 24. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17720777 # Weinreb NJ, Barranger JA, Charrow J, Grabowski GA, Mankin HJ, Mistry P: Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol. 2005 Nov;80(3):223-9. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/16247743 # Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE: Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurol. 2007 Sep;6(9):765-72. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17689147 # Moyses C: Substrate reduction therapy: clinical evaluation in type 1 Gaucher disease. Philos Trans R Soc Lond B Biol Sci. 2003 May 29;358(1433):955-60. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/12803929 # Wraith JE, Imrie J: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat. Ther Clin Risk Manag. 2009;5:877-87. Epub 2009 Nov 18. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/19956552 # McCormack PL, Goa KL: Miglustat. Drugs. 2003;63(22):2427-34; discussion 2435-6. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/14609352 (en)
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| http://linked.open...gy/drugbank/group
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| http://linked.open...drugbank/halfLife
| - The effective half-life of miglustat is approximately 6 to 7 hours. (en)
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| http://linked.open...ugbank/indication
| - For the treatment of adult patients with mild to moderate type 1 (nonneuropathic) Gaucher's disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access). Now approved in some countries for the treatment of progressive neurological symptoms in adult and pediatric patients with Niemann-Pick disease type C (NP-C). (en)
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| http://linked.open...bank/manufacturer
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| Title
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| adms:identifier
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| http://linked.open...mechanismOfAction
| - Miglustat functions as a competitive and reversible inhibitor of the enzyme glucosylceramide synthase, the initial enzyme in a series of reactions which results in the synthesis of most glycosphingolipids. The goal of treatment with miglustat is to reduce the rate of glycosphingolipid biosynthesis so that the amount of glycosphingolipid substrate is reduced to a level which allows the residual activity of the deficient glucocerebrosidase enzyme to be more effective (substrate reduction therapy), reducing the accumulation of glucocerebroside in macrophages. In vitro and in vivo studies have shown that miglustat can reduce the synthesis of glucosylceramide-based glycosphingolipids. In clinical trials, miglustat improved liver and spleen volume, as well as hemoglobin concentration and platelet count. Inhibition of glycosphingolipid synthesis has also shown to reduce intracellular lipid storage, improve fluid-phase endosomal uptake and normalize lipid transport in peripheral blood B lymphocytes of NP-C patients, which results in a decrease in the potentially neurotoxic accumulation of gnagliosides G<sub>M2</sub> and G<sub>M3</sub>, lactosylceramide and glucosylceramide, possibly preventing further neuronal damage. Other studies have also suggested that miglustat may indirectly modulate intracellular calcium homeostasis through its effects on glucosylceramide levels, and evidence has shown that an initiating factor in the pathogenesis of NP-C may be impaired calcium homeostasis related to sphingosine storage. Therefore, the effect that miglustat exerts on intracellular calcium levels may influence an important underlying pathogenic mechanism of NP-C. (en)
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| http://linked.open...drugbank/packager
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| http://linked.open...y/drugbank/patent
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| http://linked.open.../drugbank/synonym
| - Zavesca (en)
- BuDNJ (en)
- Butyldeoxynojirimycin (en)
- Miglustatum (en)
- N-(N-Butyl)deoxynojirimycin (en)
- N-Butyl deoxynojirimycin (en)
- N-Butyl-1-deoxynojirimycin (en)
- N-Butylmoranoline (en)
- NB-dnj (en)
- SC-48334 (en)
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| http://linked.open...drugbank/toxicity
| - Miglustat has been administered at doses of up to 3000 mg/day (approximately 10 times the recommended starting dose administered to Gaucher patients) for up to six months in Human Immunodeficiency Virus (HIV)-positive patients. Adverse events observed in the HIV studies included granulocytopenia, dizziness, and paresthesia. Leukopenia and neutropenia have also been observed in a similar group of patients receiving 800 mg/day or above. (en)
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| http://linked.open.../drug/hasAHFSCode
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| http://linked.open...k/foodInteraction
| - Take without regard to meals, but always at the same time and in the same way. (en)
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| http://linked.open...nk/proteinBinding
| - Miglustat does not bind to plasma proteins. (en)
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| http://linked.open...ogy/drugbank/salt
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| foaf:page
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| http://linked.open...ugbank/IUPAC-Name
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| http://linked.open...gy/drugbank/InChI
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| http://linked.open...Molecular-Formula
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| http://linked.open.../Molecular-Weight
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| http://linked.open...noisotopic-Weight
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| http://linked.open...y/drugbank/SMILES
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| http://linked.open.../Water-Solubility
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| http://linked.open...ogy/drugbank/logP
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| http://linked.open...ogy/drugbank/logS
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| http://linked.open...l/drug/hasATCCode
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| http://linked.open...nd-Acceptor-Count
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| http://linked.open...-Bond-Donor-Count
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| http://linked.open...drugbank/InChIKey
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| http://linked.open...urface-Area--PSA-
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| http://linked.open...nk/Polarizability
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| http://linked.open...bank/Refractivity
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| http://linked.open...atable-Bond-Count
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| http://linked.open...ugbank/absorption
| - Mean oral bioavailability is 97%. (en)
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| http://linked.open.../affectedOrganism
| - Humans and other mammals (en)
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| http://linked.open...casRegistryNumber
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| http://linked.open...drugbank/category
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| http://linked.open...gbank/containedIn
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| http://linked.open...k/Bioavailability
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| http://linked.open...bank/Ghose-Filter
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| http://linked.open...nk/MDDR-Like-Rule
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| http://linked.open...ank/Melting-Point
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| http://linked.open...k/Number-of-Rings
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| http://linked.open...siological-Charge
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| http://linked.open...bank/Rule-of-Five
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| http://linked.open...tional-IUPAC-Name
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| http://linked.open...strongest-acidic-
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| http://linked.open...-strongest-basic-
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