MIGLUSTAT DIPHARMA
100 mg hard capsules, currently approved in the EU and the US .
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. Treatment with Miglustat Dipharma can also reduce the progression of clinically relevant neurological symptoms in patients affected by Niemann-Pick type C disease, that are considered secondary to the abnormal accumulation of glycosphingolipids in neuronal and glial cells.
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