Migalastat

SKU: orb1709131

Description

Migalastat hydrochloride

Research Area

Metabolism Research, Pharmacology & Drug Discovery

Key Properties

• CAS Number: 75172-81-5
• MW: 199.6
• Purity: >98%
• Formula: C₆H₁₃NO₄ • HCl
• SMILES: Cl.OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O
• Target: Galactosidase
• Solubility: Soluble in DMSO (up to 50 mg/ml)

Bioactivity

• Target IC50:
  α-GalA (human):0.04 μM (Ki)|α-GalA (human):0.04 μM
• In Vivo:
  Fabry disease is an X-linked recessive disorder caused by deficient activity of alpha-galactosidase A (α-Gal A). In transgenic mice expressing the mutant human α-Gal A (TgM), oral administration of Migalastat hydrochloride (3 mg/kg per day for 4 consecutive weeks) results in a dose- and time-dependent increase in α-Gal A activity in the heart, kidneys, spleen, and liver.After a 2-week pretreatment with Migalastat hydrochloride, the half-life for all major issues is less than 1 day. Administration of Migalastat hydrochloride (100 mg/kg per day orally for 28 days) in transgenic mice leads to a reduction of 64%, 59%, and 81% in globotriaosylceramide (Gb3) levels in the kidneys, heart, and skin, respectively.
• In Vitro:
  Migalastat hydrochloride (GR181413A) demonstrates IC50 and Ki values of 0.04 μM for human lysosomal alpha-Gal A.

Storage & Handling

• Storage: -20°C
• Expiration Date: 12 months from date of receipt.
• Disclaimer: For research use only

Alternative Names

1-deoxygalactonojirimycin; DDIG; AT1001; DGJ; GR181413

Compound Identifiers

PubChem CID

11644097