1-Deoxygalactonojirimycin hydrochloride salt

CAS: 75172-81-5

Ref. 3D-MD06196

Produktinformation

• Name: 1-Deoxygalactonojirimycin hydrochloride salt
• Synonyme: (2R,3S,4R ,5S)-2-Methylolpiperidine-3,4,5-triol hydrochloride, 1-Deoxygalactonojirimycin hydrochloride, 3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride (1:1), (2R,3S,4R,5S)-, 3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride, (2R,3S,4R,5S)-, 3,4,5-Piperidinetriol, 2-(hydroxymethyl)-, hydrochloride, [2R-(2α,3α,4α,5β)]-, Galafold, Migalastat HCl, Migalastat hydrochloride

Beschreibung

Specific and potent inhibitor of lysosomal α-galactosidase with IC50 in nanomolar range. It acts as pharmacological chaperone and assists folding of the wild type and mutant versions of the enzyme. It places itself in the instable active site and prevents the damage to the enzyme during the passage through Golgi apparatus, endoplasmatic reticulum and lysosome axis. The exposure to this compound leads to increased levels of functional α-galactosidase in models for lysosomal storage disorders and brings therapeutic benefits to patients with Fabry disease.

• Marke: Biosynth

Chemische Eigenschaften

• Molekulargewicht: 199.63 g/mol
• Formel: C₆H₁₃NO₄·HCl
• Reinheit: Min. 95 Area-%
• Farbe/Form: White Powder