Migalastat HCl
Migalastat HCl, also known as AT1001 or GR181413A, is a pharmacological chaperone that selectively binds, stabilizes, and increases cellular levels of α-Gal A. Oral administration of migalastat HCl reduces tissue GL-3 in Fabry transgenic mice, and in urine and kidneys of some FD patients. Migalastat HCl may provide a potential novel genotype-specific treatment for Fabry Disease (FD). Phase 3 studies are ongoing. Fabry disease (FD) is a genetic disorder resulting from deficiency of the lysosomal enzyme α-galactosidase A (α-Gal A) which leads to globotriaosylceramide (GL-3) accumulation in multiple tissues.
| Supplier | BOC Sciences |
|---|---|
| Product # | 75172-81-5 |
| Pricing | Inquire |
| Cas | 75172-81-5 |
| Molecular Weight | 199.63 |
| Molecular Formula | C6H13NO4.HCl |
| Canonical SMILES | C1C(C(C(C(N1)CO)O)O)O.Cl |