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.

Therapeutic indications

  • Oral treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is unsuitable.
  • Treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.

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EffRx Pharmaceuticals | EffRx