UGCG and Niemann-Pick disease type C: Miglustat was dosed based on BSA and according to dosing schedule used for children with Niemann Pick disease type C, per manufacturer label [21], Miglustat is a substrate reducing agent that inhibits glucosylceramide synthase in the glycosphingolipid pathway and thereby theoretically would be anticipated to reduce production of downstream products of that pathway, including GM1- and GM2-ganglioside [21,22].