Comparative Effectiveness of Valoctocogene Roxaparvovec and Efanesoctocog Alfa in the Treatment of Severe Hemophilia A: A Matching-Adjusted Indirect Comparison of Bleeding Frequency
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DOI: 10.1007/s12325-025-03339-9
Abstract: Hemophilia A, an X-linked recessive bleeding disorder, is characterized by reduced factor VIII (FVIII) activity. Hemophilia A can significantly impact a person’s quality of life because of the risk of spontaneous bleeding. Treatment for hemophilia… read more here.
Keywords: treatment; valoctocogene roxaparvovec; efanesoctocog alfa; treated bleeds ... See more keywords
Mim8 prophylaxis in adults and adolescents with hemophilia A: 52-week efficacy and safety outcomes from the phase 3 FRONTIER2 study
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DOI: 10.1182/blood-2025-537
Abstract: Introduction Mim8 (denecimig) is a new-generation, bispecific antibody, activated factor VIII mimetic in clinical development for subcutaneous prophylaxis (PPX) for hemophilia A (HA) with or without inhibitors. The 26-week main phase of the phase 3… read more here.
Keywords: treated bleeds; main phase; extension; mim8 ... See more keywords