Indicated for: Gaucher disease; Niemann-Pick type C disease
Route of administration: oral
Substance: miglustat (enzyme inhibitor)
ATC: A16AX06 (Alimentary tract and metabolism | Other alimentary tract and metabolism products | Various alimentary tract and metabolism products)
Miglustat is a glucosylceramide synthase inhibitor used for the treatment of type 1 Gaucher disease and Niemann-Pick type C disease. It works by reducing the production of glycosphingolipids, which accumulate in cells due to these conditions.
The medication is taken orally, usually three times daily, with the dose adjusted based on the patient's weight and response to treatment. Common side effects include diarrhea, flatulence, abdominal pain, and weight loss.
Miglustat is contraindicated in patients with severe renal impairment or hypersensitivity to the active substance. Its use also requires careful monitoring of gastrointestinal function and body weight.
This medication is an important option for managing lysosomal storage disorders, helping to alleviate symptoms and slow disease progression.