Miglustat
КапсулыAn agent for the treatment of lysosomal accumulation diseases, an inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of most glycosphingolipids
Indications
Treatment of mild to moderate Gaucher disease type 1, including in case of intolerance or inability to use enzyme replacement therapy. Treatment of progressive neurological symptoms in adults and children with Niemann-Pick disease type C.
Contraindications
Age of patients with Gaucher disease under 18 years and over 70 years (insufficient clinical experience), age of patients with Niemann-Pick disease under 4 years (insufficient clinical experience), pregnancy, lactation, hypersensitivity to miglustat.
Usage
It is taken orally, regardless of food intake. The daily dose is 100-200 mg. The frequency of administration depends on the indication, age and renal function.
At CK 50-70 ml/min, the dose should not exceed 100 mg 2 times/day for patients with Gaucher disease type 1 and 200 mg 2 times/day for patients with Niemann-Pick disease type C (calculated according to body surface area for patients under 12 years of age).
At CK 30-50 ml/min the dose is 100 mg 1 time/day for patients with Gaucher disease type 1 and 100 mg 2 times/day for patients with Niemann-Pick disease type C (calculated according to body surface area for patients under 12 years of age).
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Has contraindications. Needs specialist overview.