Migalastat hydrochloride

SKU: orb1709131

Description

Migalastat hydrochloride

Research Area

Metabolism Research

Key Properties

• CAS Number: 75172-81-5
• MW: 199.63
• Purity: 100.00%
• Formula: C₆H₁₄ClNO₄
• SMILES: Cl.OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O
• Target: glycosidase,Others
• Solubility: H2O:50 mg/mL (250.46 mM)

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: store at low temperature | Powder: -20°C for 3 years | In solvent: -80°C for 1 year | Shipping with blue ice/Shipping at ambient temperature.
• Expiration Date: 12 months from date of receipt.
• Disclaimer: For research use only

Alternative Names

GR181413A, GR181413A HCl, GR181413A hydrochloride, Migalastat HCl, Migalastat hydrochloride, α-galactosidase A

Compound Identifiers

PubChem CID

11644097