1-Deoxygalactonojirimycin Hydrochloride - CAS 75172-81-5

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.

Catalog Number: R020682

CAS Number: 75172-81-5

PubChem Substance ID:355036606

Molecular Formula: C6H13NO4 • HCl

Molecular Weight:199.6

Purity: ≥95%

* For research use only. Not for human or veterinary use.

Synonym

Synonyms(2R,3S,4R,5S)-2-Hydroxymethyl-3,4,5-piperidinetriol Hydrochloride; Migalastat Hydrochloride; 1-Deoxygalactostatin Hydrochloride;

Property

Molecular Formula: C6H13NO4 • HCl
Molecular Weight199.6
Target:Galactosidase
SolubilitySoluble in DMSO
Purity≥95%
StorageStore at -20°C

Computed Descriptor

InChIKeyZJIHMALTJRDNQI-OLALXQGDSA-N