| Company Name: |
LGM Pharma |
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1-(800)-881-8210 |
| Email: |
inquiries@lgmpharma.com |
| Company Name: |
Lynnchem |
| Tel: |
86-(0)29-85992781 17792393971 |
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info@lynnchem.com |
| Company Name: |
Novachemistry |
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44-20819178-90 02081917890 |
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info@novachemistry.com |
Migalastat manufacturers
- Migalastat
-
- $2500.00
-
2026-05-11
- CAS:108147-54-2
- Purity:
- Supply Ability: 10g
|
| | Migalastat Basic information |
| Product Name: | Migalastat | | Synonyms: | GALACTOSTATIN HCL;DGJ;DGJ, HYDROCHLORIDE;DEOXYGALACTONOJIRIMYCIN HCL;DEOXYGALACTONOJIRIMYCIN, HYDROCHLORIDE;1-DEOXYGALACTONOJIRIMYCIN HCL;1,5-DIDEOXY-1,5-IMINO-D-GALACTITOL, HYDROCHLORIDE;3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, (2R,3S,4R,5S)- | | CAS: | 108147-54-2 | | MF: | C6H13NO4 | | MW: | 163.17 | | EINECS: | | | Product Categories: | Miscellaneous Natural Products | | Mol File: | 108147-54-2.mol |  |
| | Migalastat Chemical Properties |
| Melting point | 243-245 °C | | Boiling point | 361.1±42.0 °C(Predicted) | | density | 1.456±0.06 g/cm3(Predicted) | | storage temp. | 2-8°C | | pka | 13.77±0.70(Predicted) |
| Hazard Codes | Xi | | Risk Statements | 36/37/38 | | Safety Statements | 26-36 | | WGK Germany | 3 |
| | Migalastat Usage And Synthesis |
| Uses | 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[1]. | | Definition | ChEBI: Migalastat is a member of piperidines. | | Brand name | Treatment of Fabry
disease. | | in vivo | Fabry disease is an X-linked recessive disorder caused by the deficient activity of α-galactosidase A[2].
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]. | 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] | | Dosage: | 3 mg/kg | | Administration: | Oral gavage; every day for 4 weeks | | Result: | Reduced Globotriaosylceramide (Gb3) storage remarkably in kidney of mice. |
| | 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. DOI:10.1046/j.1432-1327.2000.01457.x
[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. DOI:10.1124/jpet.108.149054
[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. DOI:10.1371/journal.pone.0057631
[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. DOI:10.1093/hmg/ddy248 |
| | Migalastat Preparation Products And Raw materials |
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