Target Validation Information
TTD ID	T21678
Target Name	Alpha-galactosidase A (GLA)
Type of Target	Successful
Drug Potency against Target	Migalastat	Drug Info	Ki = 3 nM	[3]
		Drug Info	IC50 = 3 nM	[2]
	(+)-5-deoxyadeenophorine	Drug Info	IC50 = 6400 nM	[1]
	2,5-dideoxy-2,5-imino-D-altritol	Drug Info	IC50 = 780 nM	[2]
	Beta-1-C-butenyl-1-deoxygalactonojirimycin	Drug Info	IC50 = 2000 nM	[2]
	Beta-1-C-Butyl-1-deoxygalactonojirimycin	Drug Info	IC50 = 1200 nM	[2]
Action against Disease Model	AT1001	Drug Info	Bottom panel. The average potency of AT1001 for mediating an increase in GLA activity for each cell line is shown. Potency is expressed as the negative log of the concentration at which 50% of the maxim uM effect is achieved (EC50). The dotted line represents the potency of the wild type control (-log[EC50] = 6.1 or 760 nM).
References
REF 1	Flexible synthesis and biological evaluation of novel 5-deoxyadenophorine analogues. Bioorg Med Chem Lett. 2006 Jun 15;16(12):3262-7.
REF 2	2,5-Dideoxy-2,5-imino-d-altritol as a new class of pharmacological chaperone for Fabry disease. Bioorg Med Chem. 2010 Jun 1;18(11):3790-4.
REF 3	Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease. J Pharmacol Exp Ther. 2009 Mar;328(3):723-31.

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