Migalastat, also known as AT1001 or GR181413A, is a pharmacological chaperone. Migalastat binds selectively and reversibly to the active site of alpha-galactosidase A (α-Gal A), stabilizing the mutant enzyme and facilitating its proper trafficking to lysosomes, where it can degrade accumulated globotriaosylceramide (GL-3). In vitro, migalastat has shown increased α-Gal A activity in patient-derived fibroblasts with amenable mutations—often yielding ≥1.2-fold enhancement in enzymatic activity relative to baseline.
MedKoo Cat#: 573849
Name: Migalastat free base
CAS#: 108147-54-2 (free base)
Chemical Formula: C6H13NO4
Exact Mass: 163.0845
Molecular Weight: 163.17
Elemental Analysis: C, 44.17; H, 8.03; N, 8.58; O, 39.22
The following data is based on the product molecular weight 163.17 Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which will affect the solvent volumes required to prepare stock solutions.
| Concentration / Solvent Volume / Mass | 1 mg | 5 mg | 10 mg |
|---|---|---|---|
| 1 mM | 1.15 mL | 5.76 mL | 11.51 mL |
| 5 mM | 0.23 mL | 1.15 mL | 2.3 mL |
| 10 mM | 0.12 mL | 0.58 mL | 1.15 mL |
| 50 mM | 0.02 mL | 0.12 mL | 0.23 mL |
