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- Miglustat abstract "Miglustat (OGT 918, N-butyl-deoxynojirimycin) is a drug developed by Oxford GlycoSciences and marketed by Actelion and is used primarily to treat Type I Gaucher disease (GD1). It is marketed under the trade name Zavesca. Miglustat is an imino sugar, a synthetic analogue of D-glucose and a white to off-white crystalline solid that has a bitter taste. The primary pharmacological activity of miglustat is inhibition of the enzyme glucosylceramide synthase, catalyzing the first step in the biosynthesis of glycosphingolipids (GSL), i.e., the formation of glucosylceramide (GlcCer). Reduced formation of GlcCer will lead to decreased biosynthesis of more complex GSL. This therapeutic principle, called substrate reduction therapy (SRT), may be useful in disorders of intracellular (predominantly lysosomal) accumulation of GSL either due to their deficient breakdown or intracellular transport/trafficking. Miglustat exhibits a large volume of distribution and has the capacity to access deep organs such as the brain, bone and lung.Miglustat inhibits glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids (it forms glucosylceramide and accumulates within the macrophages). Miglustat is used to treat adults with mild-to-moderate Type I Gaucher disease and it is the first treatment to be approved for patients with Niemann–Pick type C disease. Miglustat may only be used in the treatment of Type I Gaucher patients for whom enzyme replacement therapy is unsuitable and it's been approved in the European Union for the treatment of progressive neurological manifestations in adult or pediatric patients with Niemann–Pick type C disease (NPC). It has also been approved for NPC treatment in Australia, Brazil, Canada, Israel, Russia, Switzerland and Turkey but not in the United States.Type I Gaucher disease is an autosomal recessive disorder; parents are generally healthy carriers with one functional and one mutated (nonfunctioning) copy of the Gaucher disease gene, GBA. People with Type I Gaucher have a defect in the enzyme called glucocerebrosidase (also known as acid β-glucosidase) that acts on a fatty substance glucosylceramide (also known as glucocerebroside). Accumulation of glucosylceramide causes liver and spleen enlargement, changes in the bone marrow and blood, and bone disease. Treatment with miglustat is known as substrate reduction therapy (SRT). Unlike enzyme replacement therapy (ERT), which has a direct effect on the breakdown of glycosphingolipids, the concept of SRT in Gaucher disease involves reduction of the delivery of potential storage material to the macrophage system. Patients treated with miglustat for 3 years show signicant improvements in organ volumes and haematological parameters. Miglustat was effective over time and showed acceptable tolerability in patients who continued with treatment for 3 years.It is also being investigated to treat Tay–Sachs disease, particularly late-onset Tay–Sachs.In October 2007, Actelion initiated a Phase IIa proof-of-concept clinical trial with miglustat in cystic fibrosis (CF). It is the first time that miglustat is being tested in a clinical setting involving 25 CF patients affected by the specific delF508 mutation.".
- Miglustat atcPrefix "A16".
- Miglustat atcSuffix "AX06".
- Miglustat bioavailability "97.0".
- Miglustat casNumber "72599-27-0".
- Miglustat chEBI "50381".
- Miglustat drugbank "DB00419".
- Miglustat fdaUniiCode "ADN3S497AZ".
- Miglustat iupacName "(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol".
- Miglustat pubchem "51634".
- Miglustat thumbnail Miglustat.svg?width=300.
- Miglustat wikiPageID "12428263".
- Miglustat wikiPageRevisionID "606521187".
- Miglustat atcPrefix "A16".
- Miglustat atcSuffix "AX06".
- Miglustat bioavailability "97.0".
- Miglustat c "10".
- Miglustat casNumber "72599".
- Miglustat chebi "50381".
- Miglustat chembl "1029".
- Miglustat chemspiderid "46764".
- Miglustat drugbank "DB00419".
- Miglustat eliminationHalfLife "25200.0".
- Miglustat excretion "Renal, unchanged".
- Miglustat h "21".
- Miglustat hasPhotoCollection Miglustat.
- Miglustat inchi "1".
- Miglustat inchikey "UQRORFVVSGFNRO-UTINFBMNBD".
- Miglustat iupacName "-1".
- Miglustat legalUs "Rx-only".
- Miglustat licenceUs "Miglustat".
- Miglustat medlineplus "a604015".
- Miglustat metabolism "Nil".
- Miglustat molecularWeight "219.28".
- Miglustat n "1".
- Miglustat o "4".
- Miglustat pregnancyUs "X".
- Miglustat proteinBound "Nil".
- Miglustat pubchem "51634".
- Miglustat routesOfAdministration "Oral".
- Miglustat smiles "OC[C@H]1NC[C@H][C@@H][C@@H]1O".
- Miglustat stdinchi "1".
- Miglustat stdinchikey "UQRORFVVSGFNRO-UTINFBMNSA-N".
- Miglustat synonyms "15".
- Miglustat unii "ADN3S497AZ".
- Miglustat verifiedrevid "462252392".
- Miglustat width "250".
- Miglustat subject Category:Orphan_drugs.
- Miglustat subject Category:Piperidines.
- Miglustat subject Category:Transferase_inhibitors.
- Miglustat type Agent114778436.
- Miglustat type CausalAgent100007347.
- Miglustat type Drug103247620.
- Miglustat type Inhibitor114724436.
- Miglustat type Matter100020827.
- Miglustat type OrphanDrugs.
- Miglustat type PhysicalEntity100001930.
- Miglustat type Substance100020090.
- Miglustat type TransferaseInhibitors.
- Miglustat type Drug.
- Miglustat type DrugProduct.
- Miglustat type FunctionalSubstance.
- Miglustat comment "Miglustat (OGT 918, N-butyl-deoxynojirimycin) is a drug developed by Oxford GlycoSciences and marketed by Actelion and is used primarily to treat Type I Gaucher disease (GD1). It is marketed under the trade name Zavesca. Miglustat is an imino sugar, a synthetic analogue of D-glucose and a white to off-white crystalline solid that has a bitter taste.".
- Miglustat label "Miglustat".
- Miglustat label "Miglustat".
- Miglustat label "Miglustat".
- Miglustat sameAs Miglustat.
- Miglustat sameAs Miglustat.
- Miglustat sameAs m.02w57mz.
- Miglustat sameAs Q425911.
- Miglustat sameAs Q425911.
- Miglustat sameAs DB00419.
- Miglustat sameAs 51634.
- Miglustat sameAs Miglustat.
- Miglustat wasDerivedFrom Miglustat?oldid=606521187.
- Miglustat depiction Miglustat.svg.
- Miglustat isPrimaryTopicOf Miglustat.