Miglustat therapy in type 1 Gaucher disease: Clinical and safety outcomes in a multicenter retrospective cohort study

Glucocerebrosidase mutations and the pathogenesis of Parkinson disease

“…I considered a diagnosis of drug-induced tremor, particularly if there was a history of receiving substrate reduction therapy (SRT) with Miglustat, a known cause of tremor (Kuter et al, 2013). In 3/7 tremor cases, SRT had been administered previously but it should be noted that in all cases, tremor was present before the treatment commenced.…”

Treatment of Niemann–Pick Type C Disease by Histone Deacetylase Inhibitors

“…Miglustat functions as a glucosylceramide synthase inhibitor and therefore affects the glycosphingolipid pathway. It had previously been approved by the FDA for treatment of Gaucher disease, which is another lysosomal storage disorder characterized by accumulation of glucosylceramide [62].The past several years of experience with miglustat indicate that it is able to stabilize NPC patients and is likely to extend their life spans [44,61].…”

Role of Endosomes and Lysosomes in Human Disease

“…An example of this is Miglustat (N-butyldeoxynojirimycin), which is an imino sugar that inhibits glucosylceramide synthase, the first committed step in glycosphingolipid synthesis. Miglustat has been used for treatment of Gaucher disease (Kuter et al 2013).…”