.Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AX06_Miglustat.Miglustat .Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AX06_Miglustat.Miglustat| name: | Miglustat |
| ATC code: | A16AX06 | route: | oral |
| n-compartments | 1 |
Miglustat is an oral iminosugar used as a substrate reduction therapy for the treatment of type 1 Gaucher disease in adults with mild to moderate disease for whom enzyme replacement therapy is not an option. It is also approved for Niemann-Pick disease type C. It inhibits glucosylceramide synthase and thereby reduces the accumulation of glycolipid substrates.
Pharmacokinetic parameters for healthy adult volunteers after a single oral dose.