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Medchemexpress LLC Migalastat (hydrochloride) | 75172-81-5 | 99.7% | 199.63 | 25 MG
Supplier: Medchemexpress LLC 25MGHY14929A
Migalastat hydrochloride (GR181413A) is an orally active α-galactosidase A molecular chaperone. It binds to the active site of unstable mutant forms of α-galactosidase A, facilitating their transport to the lysosome. Upon dissociation in the acidic environment, Migalastat enables these mutant enzymes to regain biological activity. It exhibits an IC50 and Ki value of 0.04 μM for human α-Gal A.
- Orally active α-galactosidase A molecular chaperone
- IC50 and Ki of 0.04 μM for human α-Gal A
- Facilitates transport and restores activity of mutant α-galactosidase A enzymes
- Reduces Gb3 accumulation and lysosome volume in vitro
- Increases α-Gal A activity in heart, kidney, spleen, and liver in transgenic mice
- Reduces lyso-Gb3 levels in kidney, heart, and skin in transgenic mice
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