Gaucher disease type 1

Indication for Miglustat

Population group: Suitable for both men and women, only adults (18 - 65 years old)

Miglustat is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.

For this indication, competent medicine agencies globally authorize below treatments:

100-300 mg in 3 divided doses daily

Route of admnistration

Oral

Defined daily dose

100 - 300 mg

Dosage regimen

From 33.333 To 100 mg 3 time(s) per day every day

Loading dose

300 mg

Detailed description

The recommended starting dose for the treatment of adult patients with type 1 Gaucher disease is 100 mg three times a day.

Temporary dose reduction to 100 mg once or twice a day may be necessary in some patients because of diarrhoea.

Dosage considerations

Miglustat can be taken with or without food.

Active ingredient

Miglustat is an inhibitor of glucosylceramide synthase, the enzyme responsible for the first step in the synthesis of most glycolipids. Miglustat is indicated for the treatment of adult patients with mild to moderate type 1 Gaucher disease and for the treatment of progressive neurological manifestations in patients with Niemann-Pick type C disease.

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