A single intravenous infusion of lonvoguran ziclumeran reduces hereditary angioedema attack rates by 87% compared to placebo in a phase 3 trial.
Hemophilia Treatment and Research
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FXI-targeting monoclonal antibodies amrecibart and cenvacibart inhibit intrinsic pathway-triggered coagulation without affecting extrinsic pathway in humans and primates.
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One-stage assay FVIII activity accurately reflects AAV-FVIII-SQ function and better predicts bleeding outcomes in hemophilia A gene therapy than chromogenic assays.
AAV gene therapy BBM-H901 is safe and reduces bleeding episodes in 11 adolescents with hemophilia B, with FIX activity reaching an average of 41.8 IU/dl at 52 weeks.
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Topics moving, week to 23 Oct 2026
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