Hepatology
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See the full rubric →Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.
Clinical takeawayConsider BBM-H901 gene therapy for adolescents with severe or moderately severe hemophilia B (FIX:C ≤2 IU/dl) in clinical trials, monitoring for adverse events such as liver enzyme elevation and corticosteroid-associated rash.
What it foundAAV gene therapy BBM-H901 in adolescents with hemophilia B (FIX:C ≤2 IU/dl) increased mean FIX:C to 41.8 IU/dl at 52 weeks and reduced annualized bleeding rate from 13.9 to 0.5, with observed adverse events including elevated liver enzymes, white blood cell count, and rash.
ContextExtends prior adult data on AAV gene therapy for hemophilia B to adolescents, showing similar efficacy and safety, but with notable adverse events requiring careful monitoring.
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Xue F … Zhang L · Nature Medicine · IF 52.5 · PubMed ↗