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Miglustat Transferase inhibitor

Cat.No.E1029

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

Miglustat Transferase inhibitor Chemical Structure

Chemical Structure

Molecular Weight: 219.28

Quality Control

Chemical Information, Storage & Stability

Molecular Weight 219.28 Formula

C10H21NO4

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

Solubility

In vitro
Batch:

DMSO : 44 mg/mL (200.65 mM)
(Moisture-contaminated DMSO may reduce solubility. Use fresh, anhydrous DMSO.)

Water : 44 mg/mL

Ethanol : 22 mg/mL

Molarity Calculator

Mass Concentration Volume Molecular Weight

In vivo
Batch:

In vivo Formulation Calculator (Clear solution)

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Working concentration: mg/ml;

Method for preparing DMSO master liquid: mg drug pre-dissolved in μL DMSO ( Master liquid concentration mg/mL, Please contact us first if the concentration exceeds the DMSO solubility of the batch of drug. )

Method for preparing in vivo formulation: Take μL DMSO master liquid, next addμL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O, mix and clarify.

Method for preparing in vivo formulation: Take μL DMSO master liquid, next add μL Corn oil, mix and clarify.

Note: 1. Please make sure the liquid is clear before adding the next solvent.
2. Be sure to add the solvent(s) in order. You must ensure that the solution obtained, in the previous addition, is a clear solution before proceeding to add the next solvent. Physical methods such
as vortex, ultrasound or hot water bath can be used to aid dissolving.

Mechanism of Action

Targets/IC50/Ki
ceramide glucosyltransferase [1]
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 this compound.[2]

In vivo

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

References

Clinical Trial Information

(data from https://clinicaltrials.gov, updated on 2024-05-22)

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

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