Molfile
Inchi
Smiles
Synonyms:
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migalastat HCl
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GR181413A
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1-Deoxygalactonojirimycin hydrochloride
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1-Deoxygalactonojirimycin
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migalastat
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migalastat hydrochloride
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galafold
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Migalastat is a pharmacological chaperone that is designed to selectively and reversibly bind with high affinity to the active sites of certain mutant forms of alpha-Gal A, the genotypes of which are referred to as amenable mutations. Migalastat binding stabilizes these mutant forms of alpha-Gal A in the endoplasmic reticulum and facilitates their proper trafficking to lysosomes where dissociation of migalastat restores alpha-Gal A activity, leading to the catabolism of GL-3 and related substrates.
- Molecular weight: 163.17
- Formula: C6H13NO4
- CLOGP: -0.40
- LIPINSKI: 0
- HAC: 5
- HDO: 5
- TPSA: 92.95
- ALOGS: 0.50
- ROTB: 1
- Status: ONP
Legend: OFP - off patent OFM - off market ONP - on patent
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