Chemical formula: C₁₀H₂₁NO₄ Molecular mass: 219.278 g/mol PubChem compound: 51634
Miglustat is indicated for:
Population group: 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 (click for details):
Population group: only children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 - 65 years old)
Miglustat is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.
For this indication, competent medicine agencies globally authorize below treatments (click for details):
Population group: only adults (18 years old or older)
Miglustat is an enzyme stabiliser of cipaglucosidase alfa long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).
For this indication, competent medicine agencies globally authorize below treatments (click for details):
Miglustat is contraindicated in the following cases:
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