Valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, enables endogenous factor VIII (FVIII) expression and confers bleed control. To present the final 5-year efficacy and safety results...
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Hemophilia A and B are inherited bleeding disorders caused by deficiencies of coagulation factors VIII and IX. The development of neutralizing alloantibodies (inhibitors) remains the most serious treatment...
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Emicizumab is an established prophylactic therapy for haemophilia A (HA). However, real-world multicentre evidence on long-term outcomes, particularly in a resource-constrained setting, remains limited....
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Hemophilia A (HA) management has progressed significantly, yet standard and extended half-life (EHL) clotting factors present persistent limitations, including intravenous burden and suboptimal trough levels. This narrative review highlights recently approved agents and phase III candidates for HA. In replacement therapy, efanesoctocog alfa maintains normal-to-near-normal factor VIII (FVIII) levels weekly by bypassing endogenous von Willebrand factor...
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