Miglustat is an inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of most glycolipids. The inhibitory action on glucosylceramide synthase forms the rationale for substrate reduction therapy in Gaucher disease. Miglustat Dipharma is also indicated to reduce the progression of clinically relevant neurological symptoms in patients affected by Niemann-Pick type C disease, which are considered secondary to the abnormal accumulation of glycosphingolipids in neuronal and glial cells.

EffRx is Dipharma’s distributor for Miglustat Dipharma in Switzerland.

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