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Synonyms: 1-Deoxygalactonojirimycin | Galafold® | GR-181413A | GR181413A
migalastat is an approved drug (EMA (2016), FDA (2018))
Compound class: Synthetic organic
Comment: Migalastat was developed by Amicus Therapeutics as a first-in-class oral monotherapy for the treatment of Fabry disease . Mechanistically it acts as a pharmacological chaperone that stabilises certain mutant forms of α-galactosidase that are found in Fabry disease patients and enhances enzyme activity . It is a stereoisomer of 1-deoxynojirimycin.
Ligand Activity Visualisation Charts
These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.✖
1. Asano N, Ishii S, Kizu H, Ikeda K, Yasuda K, Kato A, Martin OR, Fan JQ. (2000)
In vitro inhibition and intracellular enhancement of lysosomal alpha-galactosidase A activity in Fabry lymphoblasts by 1-deoxygalactonojirimycin and its derivatives.
Eur J Biochem, 267 (13): 4179-86. [PMID:10866822]
2. Hughes DA, Nicholls K, Shankar SP, Sunder-Plassmann G, Koeller D, Nedd K, Vockley G, Hamazaki T, Lachmann R, Ohashi T et al.. (2017)
Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study.
J Med Genet, 54 (4): 288-296. [PMID:27834756]