In the European Union, miglustat (Opfolda), in combination with cipaglucosidase alfa, is a long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α‑glucosidase [GAA] deficiency).

ZAVESCA ® is a prescription medicine used alone to treat adults with mild to moderate type 1 Gaucher disease. ZAVESCA ® is used only in people who cannot be treated with enzyme replacement therapy. It is not known if ZAVESCA ® is safe and effective in children under 18 years of age. What should I tell my doctor before taking ZAVESCA®?

Miglustat works by stopping the body from making glucosylceramide. Miglustat is used in combination with cipaglucosidase alfa-atga injection (Pombiliti™) to treat late-onset of Pompe disease in patients who are not improving on their current enzyme replacement therapy (ERT).

2024年2月8日 · What is miglustat? Miglustat is used to treat mild to moderate type 1 Gaucher disease in adults who cannot receive enzyme replacement therapy. Miglustat may also be used for purposes not listed in this medication guide.

Miglustat is a glucosylceramide synthase inhibitor indicated as monotherapy for treatment of adult patients with mild/moderate type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (1.1). Recommended dosage is 100 mg administered orally three times a day at regular intervals (2.1).

Miglustat is used to treat Gaucher disease type 1 (a condition in which a certain fatty substance is not broken down normally in the body and instead builds up in some organs and causes liver, spleen, bone, and blood problems).

Miglustat is used for the long-term treatment of a certain rare genetic problem (Gaucher's disease). It works by decreasing the amount of a certain natural fatty substance...

2023年10月9日 · Miglustat is a medication used as a monotherapy to treat type 1 Gaucher disease in adults (Zavesca brand) and in combination with cipaglucosidase alfa in the treatment of Pompe disease in adults (Opfolda brand).

2022年3月15日 · Miglustat (Zavesca) is an oral medication for Gaucher disease. It works by blocking glucosylceramide from being made. This then lowers the amount of glucosylceramide in your body so that it can't build up.

2024年4月24日 · Generic name: miglustat [ mi-GLOO-stat ] Drug class: Miscellaneous metabolic agents. Medically reviewed by Philip Thornton, DipPharm. Last updated on Apr 24, 2024. Uses; Warnings; Before taking; Dosage; Side effects; Interactions; What is Zavesca? Zavesca reduces the formation of a certain protein in the body in people with type 1 Gaucher disease.