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fidanacogene elaparvovec-dzkt (Beqvez)

Indications: - adenovirus-associated vector-based gene therapy indicated for treatment of adults with moderate to severe hemophilia B Dosage: - 5 × 1011 vector genomes per kg - IV infusion after dilution in 0.9%sodium chloride with 0.25% human serum albumin, final volume of 200 mL over ~60 minutes Adverse effects: - hepatotoxicty - hepatocellular carcinoma? - increased serum transaminases

General

gene therapy

References

  1. HIGHLIGHTS OF PRESCRIBING INFORMATION fidanacogene elaparvovec-dzkt (Beqvez) for intravenous infusion https://labeling.pfizer.com/ShowLabeling.aspx?id=20452&format=pdf