Migalastat hydrochloride
Based on 2 publication(s) in Google Scholar
Migalastat (GR181413A free base) hydrochloride is an orally active α-galactosidase A molecular chaperone, with an IC50 value of 0.04 μM for human α-Gal A. Migalastat binds to the active site of certain unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. After dissociation in the acidic environment, Migalastat enables the mutant α-galactosidase A to exhibit biological activity.
For research use only. We do not sell to patients.
- Purity: 99.70%
- CAS No.: 75172-81-5
- Formula: C6H14ClNO4
- Molecular Weight:199.63
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Storage:
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
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Biological Activity
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Chemical Information
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Solvent & Solubility
- Purity & Documentation
- References
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Help & FAQs
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Publications Citing Use of MedChemExpress (MCE) Migalastat hydrochloride
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Biological Activity
IC50: 0.04 μM (human α-Gal A)[1]; Ki: 0.04 μM (human α-Gal A)[1]
Both IC50 and Ki values of Migalastat hydrochloride (GR181413A) toward human lysosomal a-Gal A are 0.04 μM[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Cell Line:EHK cells mutated α-Gal A
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Concentration:10 μM
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Incubation Time:9 days
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Result:Reduced Gb3 accumulation and lysosome volume.
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Male nontransgenic (Non-Tg) C57BL/6 mice; transgenic mice expressing human mutant R301Q α-Gal A (TgM), α-Gal A knockout mice (KO), mice express human R301Q α-Gal A in a null background (TgM/KO)[2]
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Dosage:3 mg/kg
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Administration:Oral gavage; every day for 4 weeks
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Result:Reduced Globotriaosylceramide (Gb3) storage remarkably in kidney of mice.
Chemical Information
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CAS No. 75172-81-5
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Appearance Solid
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Molecular Weight 199.63
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Formula C6H14ClNO4
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Color White to light brown
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SMILES
O[C@H]1[C@@H](CO)NC[C@H](O)[C@H]1O.Cl
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Synonyms
GR181413A
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
4°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Publications (2)
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Journal Impact Factor
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Most Recent
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Int J Biol Macromol
Identification of c.146G > A mutation in a Fabry patient and its correction by customized Cas9 base editors in vitro. [Abstract]2024 Dec;282(Pt 3):136922. PMID: 39490876 -
Open Heart
Novel GLA variant in Fabry cardiomyopathy: evidence of pathogenicity and amenability to migalastat. [Abstract]2026 May 20;13(1):e004016. PMID: 42161426
Solvent & Solubility
H2O : ≥ 200 mg/mL (1001.85 mM)
* "≥" means soluble, but saturation unknown.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
For the following dissolution methods, please prepare the working solution directly:
It is recommended to prepare fresh solutions and use them promptly within a short period of time.
The percentages shown for the solvents indicate their volumetric ratio in the final prepared solution. If precipitation or phase separation occurs during preparation, heat and/or sonication can be used to aid dissolution.
Add each solvent one by one: PBS
Solubility: 100 mg/mL (500.93 mM); Clear solution; Need ultrasonic
Purity & Documentation
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Data Sheet (278 KB)
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SDS (393 KB)
- English - EN (393 KB)
- Français - FR (393 KB)
- Deutsch - DE (393 KB)
- Norwegian - NO (393 KB)
- Español - ES (393 KB)
- Swedish - SV (393 KB)
- Italian - IT (393 KB)
- Korean - KR (393 KB)
- Portuguese - PT (393 KB)
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Handling Instructions (2659 KB)
References
[1]. Asano N, et al. In vitro inhibition and intracellular enhancement of lysosomal alpha-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives. Eur J Biochem. 2000 Jul;267(13):4179-86. [Content Brief]
[2]. Ishii S, et al. Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease. J Pharmacol Exp Ther. 2009 Mar;328(3):723-31. [Content Brief]
[3]. Young-Gqamana B, et al. Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients. PLoS One. 2013;8(3):e57631. [Content Brief]
[4]. Welford RWD, et al. Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types. Hum Mol Genet. 2018 Oct. 27(19):3392-3403. [Content Brief]
Complete Stock Solution Preparation Table
Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture). When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.
| Optional Solvent | Concentration Solvent Mass | 1 mg | 5 mg | 10 mg | 25 mg |
|---|---|---|---|---|---|
| H2O | 1 mM | 5.0093 mL | 25.0463 mL | 50.0927 mL | 125.2317 mL |
| 5 mM | 1.0019 mL | 5.0093 mL | 10.0185 mL | 25.0463 mL | |
| 10 mM | 0.5009 mL | 2.5046 mL | 5.0093 mL | 12.5232 mL | |
| 15 mM | 0.3340 mL | 1.6698 mL | 3.3395 mL | 8.3488 mL | |
| 20 mM | 0.2505 mL | 1.2523 mL | 2.5046 mL | 6.2616 mL | |
| 25 mM | 0.2004 mL | 1.0019 mL | 2.0037 mL | 5.0093 mL | |
| 30 mM | 0.1670 mL | 0.8349 mL | 1.6698 mL | 4.1744 mL | |
| 40 mM | 0.1252 mL | 0.6262 mL | 1.2523 mL | 3.1308 mL | |
| 50 mM | 0.1002 mL | 0.5009 mL | 1.0019 mL | 2.5046 mL | |
| 60 mM | 0.0835 mL | 0.4174 mL | 0.8349 mL | 2.0872 mL | |
| 80 mM | 0.0626 mL | 0.3131 mL | 0.6262 mL | 1.5654 mL | |
| 100 mM | 0.0501 mL | 0.2505 mL | 0.5009 mL | 1.2523 mL |
* Note: If you choose water as the stock solution, please dilute it to the working solution, then filter and sterilize it with a 0.22 μm filter before use.
Migalastat hydrochloride Related Classifications
HY-14929A Related Classifications
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