Afegostat (hydrochloride)

Afegostat hydrochloride (D-Isofagomine hydrochloride) is a potent β-galactosidase inhibitor with activity ameliorating GM1-gangliosidosis and Morquio B disease-associated mutations. Afegostat hydrochloride is able to induce the maturation of mutant β-galactosidase in fibroblasts from patients with GM1-gangliosidosis. Afegostat hydrochloride also promotes the reduction of keratin sulfate and oligosaccharide load in patient cells.