Miglustat
- Generic Name
- Miglustat
- Brand Names
- Opfolda, Zavesca, Miglustat Dipharma, Miglustat Gen.Orph, Yargesa
- Drug Type
- Small Molecule
- Chemical Formula
- C10H21NO4
- CAS Number
- 72599-27-0
- Unique Ingredient Identifier
- ADN3S497AZ
- Background
Miglustat, commonly marketed under the trade name Zavesca, is a drug used to treat Gaucher disease. It inhibits the enzyme glucosylceramide synthase, an essential enzyme for the synthesis of most glycosphingolipids. It is only used for patients who cannot be treated with enzyme replacement therapy with imiglucerase. Miglustat is now the first and only approv...
- Indication
For the treatment of adult patients with mild to moderate type 1 (nonneuropathic) Gaucher's disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity, or poor venous access). Now approved in some countries for the treatment of progressive neurological symptoms in adult and pediatric pat...
- Associated Conditions
- Mild, moderate Gaucher Disease, Type 1
- Associated Therapies
- -
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Drug Development Updates
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