Afegostat HCl

Afegostat is a potent acid β-glucosidase (GCase) pharmacological chaperone, inhibiting both wild-type and mutant enzymes with nanomolar affinity (K (i) ~30 nM) . It has been investigated in preclinical research for Gaucher disease, including in vitro enzyme studies and in vivo animal models to assess its potential in stabilizing and enhancing mutant GCase activity.