Migalastat
Based on 2 publication(s) in Google Scholar
Migalastat (GR181413A free base) 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.
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- CAS No.: 108147-54-2
- Formule: C6H13NO4
- Masse moléculaire:163.17
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Stockage:
Please store the product under the recommended conditions in the Certificate of Analysis.
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Activité biologique
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Chemical Information
- Pureté et documentation
- Références
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Publications Citing Use of MedChemExpress (MCE) Migalastat
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Activité biologique
IC50: 0.04 μM (human α-Gal A)[1]; Ki: 0.04 μM (human α-Gal A)[1]
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Cell Line
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Type | Value | Description | References |
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| Lymphocyte | CC50 |
>200 μM
Compound: 2; DGJ
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Cytotoxicity against human lymphocytes assessed as reduction in cell viability after 48 to 72 hrs by alamar blue assay
Cytotoxicity against human lymphocytes assessed as reduction in cell viability after 48 to 72 hrs by alamar blue assay
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[PMID: 27474919] |
| Lymphocyte | CC50 |
>200 μM
Compound: DGJ
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Cytotoxicity against human lymphocytes measured after 48 to 72 hrs by alamar blue assay
Cytotoxicity against human lymphocytes measured after 48 to 72 hrs by alamar blue assay
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[PMID: 27744182] |
Migalastat inhibits human lysosomal a-Gal A with IC50 and Ki values of 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.
Migalastat (oral gavage, 3 mg/kg daily for 4 weeks) increases α-Gal A activity in heart, kidney, spleen, and liver in a dose- and time-dependently in transgenic mice that express human mutant alpha-Gal A (TgM)[2].
Migalastat shows the half-life of less than 1 day in all major issues in TgM for 2 weeks pretreatment[2].
Migalastat (oral gavage, 100 mg/kg daily for 28 days) to transgenic mice reduces lyso-Gb3 levels up to 64%, 59%, and 81% in kidney, heart, and skin, respectively[3].
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.
| NCT Number | Sponsor | Condition | Start Date |
Phase
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|---|---|---|---|---|
| NCT01329991 | Plexxikon| | 2011-05 | PHASE1 |
Chemical Information
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CAS No. 108147-54-2
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Masse moléculaire 163.17
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Formule C6H13NO4
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SMILES
O[C@H]1[C@@H](CO)NC[C@H](O)[C@H]1O
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Synonyms
GR181413A free base; 1-Deoxygalactonojirimycin
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Livraison
Room temperature in continental US; may vary elsewhere.
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Stockage
Please store the product under the recommended conditions in the Certificate of Analysis.
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 Oct 25:136922. 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
Pureté et documentation
Références
[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]
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
Migalastat Related Classifications
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