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Information about Imiglucerase

Imiglucerase (im" i gloo' ser ase) was the first recombinant form of glucocerebrosidase approved for therapy of type 1 Gaucher disease. Imiglucerase is prepared by recombinant techniques using Chinese hamster ovary cells. It differs from the native enzyme in one amino acid (histidine at position 495 instead of arginine) and by modification of the glycosylation sites so that they terminate in mannose sugars, which are specifically recognized and taken up by macrophages.

FDA approval information for Imiglucerase

Imiglucerase was approved for use in the United States in 1994 and soon became the most widely used enzyme replacement therapy for Gaucher disease. It is available as a lyophilized powder in vials of 200 and 400 Units.

Dosage and administration for Imiglucerase

The typical dose is 60 Units/kg given by intravenous infusion over 1 to 2 hours every two weeks.

Side effects of Imiglucerase

Side effects are uncommon and generally mild, but can include local infusion site reactions, fatigue, headache, dizziness, abdominal pain, nausea, diarrhea, back pain, fever and rash. Rare, but potentially severe adverse reactions include hypersensitivity reactions and anaphylaxis.

genetic disorder agents

cystic fibrosis agents

enzyme replacement therapy

glucosylceramide synthase inhibitors (substrate restriction therapy)

lysosomal acid lipase deficiency agents


homocystinuria agents

Huntington disease agents

Monoclonal Antibodies

Tyrosinemia Agents

Urea Cycle Disorder Agents

Hematologic Agents

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