108147-54-2
Chemical Structure
Migalastat
Synonym(s): GR181413A free base; 1-Deoxygalactonojirimycin
- CAS No.: 108147-54-2
- Formula:C6H13NO4
- Molecular Weight:163.17
IUPAC Name: (2R,3S,4R,5S)-2-(hydroxymethyl)piperidine-3,4,5-triol
InChIKey: LXBIFEVIBLOUGU-DPYQTVNSSA-N
SMILES: O[C@H]1[C@@H](CO)NC[C@H](O)[C@H]1O
Biological Activity: 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].
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Migalastat | 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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Migalastat-d5 | Migalastat-d5 (GR181413A-d5 (free base)) is deuterium labeled Migalastat. 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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