1-Deoxynojirimycin (hydrochloride)

1-Deoxynojirimycin (1-dNM) (hydrochloride), produced by Bacillus species, is a glucose analog that potently inhibits α-glucosidase I and II. It prevents the formation of complex N-linked oligosaccharides in yeast and intact mammalian cells by inhibiting both α-glucosidase I and II with IC50 values of ~2 µM. The FDA has granted orphan drug designation to dNM (hydrochloride) for the treatment of Pompe disease, an inherited lysosomal storage disorder caused by a mutation that alters the structure and stability of α-glucosidase. 1-dNM, at a concentration of 0.4 mM, also inhibits virus spread in HIV-infected lymphocyte cultures by interfering with α-glucosidase activity.