| Size | Price | Stock | Qty |
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| 10mg |
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| 25mg |
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| 50mg |
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| 100mg |
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| 250mg |
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| Other Sizes |
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Miglustat HCl (OGT-918; NB-DNJ; Zavesca) is an FDA approved drug used to treat Type I Gaucher disease (GD1). 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 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.
| ln Vitro |
In cystic fibrosis (CF) folding epithelial IB3-1 and CuFi-1 cells, the function of F508del-CFTR (cystic fibrosis transmembrane conductance regulator) is restored by imiglustat hydrochloride (200 μM; 2, 4, and 24 hours). Key reactions of both CF and non-CF cells to Pseudomonas aeruginosa are diminished by miglustat hydrochloride [1].
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|---|---|
| ln Vivo |
The oral medication miglustat hydrochloride (0.2 mg/kg; once) corrects synaptic plasticity deficits, reinstates ERK activation, and reacts to hyperexcitability [2].
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| Animal Protocol |
Animal/Disease Models: NPC1−/− mice[1]
Doses: 0.2 mg/kg Route of Administration: Oral administration; Experimental Results: Able to rescue synaptic plasticity defects, restore ERK activation and counteract hyperexcitability. |
| References | |
| Additional Infomation |
See also: Miglustat (annotation moved to).
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| Molecular Formula |
C10H21NO4
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|---|---|
| Molecular Weight |
255.7390
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| Exact Mass |
255.123
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| CAS # |
210110-90-0
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| Related CAS # |
Miglustat;72599-27-0;Miglustat-d9 hydrochloride;1883545-57-0
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| PubChem CID |
6603107
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| Appearance |
White to light brown solid powder
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| Boiling Point |
421.2ºC at 760 mmHg
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| Melting Point |
169-172ºC
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| Flash Point |
208.5ºC
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| Vapour Pressure |
7.37E-09mmHg at 25°C
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| Hydrogen Bond Donor Count |
5
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| Hydrogen Bond Acceptor Count |
5
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| Rotatable Bond Count |
4
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| Heavy Atom Count |
16
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| Complexity |
190
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| Defined Atom Stereocenter Count |
4
|
| SMILES |
CCCCN1C[C@@H]([C@H]([C@@H]([C@H]1CO)O)O)O.Cl
|
| InChi Key |
QPAFAUYWVZMWPR-ZSOUGHPYSA-N
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| InChi Code |
InChI=1S/C10H21NO4.ClH/c1-2-3-4-11-5-8(13)10(15)9(14)7(11)6-12/h7-10,12-15H,2-6H2,1H31H/t7-,8+,9-,10-/m1./s1
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| Chemical Name |
(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride
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| Synonyms |
OGT918 OGT 918 OGT-918 N-butyldeoxynojirimycin NB-DNJ Miglustat hydrochloride Zavesca.
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| HS Tariff Code |
2934.99.9001
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| Storage |
Powder -20°C 3 years 4°C 2 years In solvent -80°C 6 months -20°C 1 month Note: Please store this product in a sealed and protected environment (e.g. under nitrogen), avoid exposure to moisture. |
| Shipping Condition |
Room temperature (This product is stable at ambient temperature for a few days during ordinary shipping and time spent in Customs)
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| Solubility (In Vitro) |
DMSO : ~65 mg/mL (~254.16 mM)
H2O : ≥ 34 mg/mL (~132.95 mM) |
|---|---|
| Solubility (In Vivo) |
Solubility in Formulation 1: ≥ 3.25 mg/mL (12.71 mM) (saturation unknown) in 10% DMSO + 40% PEG300 + 5% Tween80 + 45% Saline (add these co-solvents sequentially from left to right, and one by one), clear solution.
For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 32.5 mg/mL clear DMSO stock solution to 400 μL of PEG300 and mix evenly; then add 50 μL of Tween-80 to the above solution and mix evenly; then add 450 μL of normal saline to adjust the volume to 1 mL. Preparation of saline: Dissolve 0.9 g of sodium chloride in 100 mL ddH₂ O to obtain a clear solution. Solubility in Formulation 2: ≥ 3.25 mg/mL (12.71 mM) (saturation unknown) in 10% DMSO + 90% (20% SBE-β-CD in Saline) (add these co-solvents sequentially from left to right, and one by one), clear solution. For example, if 1 mL of working solution is to be prepared, you can add 100 μL of 32.5 mg/mL clear DMSO stock solution to 900 μL of 20% SBE-β-CD physiological saline solution and mix evenly. Preparation of 20% SBE-β-CD in Saline (4°C，1 week): Dissolve 2 g SBE-β-CD in 10 mL saline to obtain a clear solution. View More
Solubility in Formulation 3: ≥ 3.25 mg/mL (12.71 mM) (saturation unknown) in 10% DMSO + 90% Corn Oil (add these co-solvents sequentially from left to right, and one by one), clear solution. Solubility in Formulation 4: 100 mg/mL (391.02 mM) in PBS (add these co-solvents sequentially from left to right, and one by one), clear solution; with ultrasonication. |
| Preparing Stock Solutions | 1 mg | 5 mg | 10 mg | |
| 1 mM | 3.9102 mL | 19.5511 mL | 39.1022 mL | |
| 5 mM | 0.7820 mL | 3.9102 mL | 7.8204 mL | |
| 10 mM | 0.3910 mL | 1.9551 mL | 3.9102 mL |
*Note: Please select an appropriate solvent for the preparation of stock solution based on your experiment needs. For most products, DMSO can be used for preparing stock solutions (e.g. 5 mM, 10 mM, or 20 mM concentration); some products with high aqueous solubility may be dissolved in water directly. Solubility information is available at the above Solubility Data section. Once the stock solution is prepared, aliquot it to routine usage volumes and store at -20°C or -80°C. Avoid repeated freeze and thaw cycles.
Calculation results
Working concentration： mg/mL；
Method for preparing DMSO stock solution： mg drug pre-dissolved in μL DMSO (stock solution 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 stock solution, next add μL PEG300, mix and clarify, next addμL Tween 80, mix and clarify, next add μL ddH2O，mix and clarify.
(1) Please be sure that the solution is clear before the addition of next solvent. Dissolution methods like vortex, ultrasound or warming and heat may be used to aid dissolving.
(2) Be sure to add the solvent(s) in order.
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