Miglustat

Synonyms: Zavesca, N‐butyldeoxynojirimycin, OGT 918

Miglustat (Zavesca, N‐butyldeoxynojirimycin, OGT 918) is an orally administered ceramide glucosyltransferase inhibitor which prevents the lysosomal accumulation of glucocerebroside.

Miglustat Chemical Structure

Miglustat Chemical Structure

CAS No. 72599-27-0

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Miglustat Related Products

Biological Activity

Description

Miglustat (Zavesca, N‐butyldeoxynojirimycin, OGT 918) is an orally administered ceramide glucosyltransferase inhibitor which prevents the lysosomal accumulation of glucocerebroside.

Targets
ceramide glucosyltransferase [1]
In vitro
In vitro

Both HCT116 and Lovo cells show decreased tumor microsphere growth with pronounced growth retardation of Lovo cell spheroids at low concentrations of Miglustat. A marked elevation of SM and PC in is observed in Lovo cells treated with Miglustat.[2]

Cell Research Cell lines HCT116, Lovo cells
Concentrations 100 µM
Incubation Time 4 days
Method

Lovo and HCT116 cells are cultivated in MEM-alpha medium, cells are cultivated in 10 mL culture medium in- or without the presence of 100 µM Miglustat. Cells are cultivated in 10 cm tissue culture dishes for 4 days. Cells are counted, split and treated for another 2 days, each in triplicates. Cells are harvested and used for FACS-, protein-, and TLC-analysis.

In Vivo
In vivo

Miglustat treatment of Mcoln1−/− mice successfully delays impairments in cerebellar-mediated motor coordination, Purkinje cell death, and cerebellar microgliosis.[3]

Animal Research Animal Models Mcoln1−/− mice
Dosages 300 mg/kg
Administration i.p.
NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT05174039 Active not recruiting
Batten Disease
Beyond Batten Disease Foundation|Theranexus
February 2 2022 Phase 1|Phase 2
NCT02185651 Terminated
Pompe Disease|Hypersensitivity Reaction
University of Florida|Amicus Therapeutics
October 2016 Phase 1
NCT00194649 Completed
Contraception
University of Washington|Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
June 2005 Phase 4
NCT00418847 Completed
Gangliosidoses GM2
The Hospital for Sick Children|Actelion
July 2004 Phase 2

Chemical Information & Solubility

Molecular Weight 219.28 Formula

C10H21NO4

CAS No. 72599-27-0 SDF --
Storage (From the date of receipt) 3 years -20°C powder

In vitro
Batch:

DMSO : 44 mg/mL ( (200.65 mM) Moisture-absorbing DMSO reduces solubility. Please use fresh DMSO.)

Water : 44 mg/mL

Ethanol : 22 mg/mL


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In vivo
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Tech Support

Answers to questions you may have can be found in the inhibitor handling instructions. Topics include how to prepare stock solutions, how to store inhibitors, and issues that need special attention for cell-based assays and animal experiments.

Handling Instructions

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