Olivier D. Christophe

Small interfering RNA targeting protein Z-dependent protease inhibitor improves hemostasis in Von Willebrand factor- and Factor X-deficient mice

Non-factor therapeutics have transformed the treatment paradigm in hemophilia A. Owing to their mode of action, these agents may also provide benefit for other rare bleeding disorders (RBDs) such as von Willebrand disease (VWD)-type 3 and Factor X (FX) deficiency. Targeting prote...