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What is fabrazyme?

See the DrugPatentWatch profile for fabrazyme

Fabrazyme is the brand name for agalsidase beta, a recombinant enzyme used to treat Fabry disease. It’s an enzyme replacement therapy that provides human alpha-galactosidase A, the enzyme that people with Fabry disease are deficient in. By supplying this enzyme, Fabrazyme helps break down globotriaosylceramide (Gb3) that accumulates in cells and tissues.

Key points:
- Indication: treating Fabry disease in both adults and pediatric patients to reduce Gb3 accumulation and help manage symptoms and organ involvement.
- Dosing: usually given by intravenous infusion every 2 weeks at 1 mg/kg body weight (infusion typically lasts 1–2 hours).
- Safety: common infusion reactions (fever, chills, nausea, headache), possible antibody development, rare allergic reactions. Contraindicated in people with a known hypersensitivity to agalsidase beta or any excipients.
- Notes: not a cure, but can slow disease progression and help manage symptoms. Storage: refrigerate (2–8°C), protected from light.

If you’re considering this for yourself or someone else, a healthcare provider can give detailed dosing, monitoring, and safety information.



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