Miglustat Gen.Orph

Miglustat Gen.Orph is used to treat two inherited diseases that affect the way the body handles fats. Both diseases cause a build-up of fatty substances called glycosphingolipids in the body. Miglustat Gen.Orph is used to treat the following patients: - adults (aged 18 years and above) with mild to moderate type-1 Gaucher disease. Patients with this disease lack an enzyme called glucocerebrosidase, which results in a glycosphingolipid called glucosylceramide building up in different parts of the body, such as the spleen, liver and bones. Miglustat Gen.Orph is used in patients who cannot receive the standard treatment of enzyme-replacement therapy (ERT); - patients of all ages with Niemann-Pick type-C disease, a potentially fatal disease in which glycosphingolipids build up within cells in the brain and elsewhere in the body. Miglustat Gen.Orph is used to treat the neurological symptoms of the disease (symptoms affecting the brain and nerves). These include loss of coordination, problems with ‘saccadic’ (rapid) eye movements that can lead to impaired vision, delayed development, difficulty swallowing, decreased muscle tone, fits and learning difficulties. Miglustat Gen.Orph is a ‘generic medicine’. This means that Miglustat Gen.Orph contains the same active substance and works in the same way as a ‘reference medicine’ already authorised in the EU called Zavesca.