La dernière actualité médicale HaemophiliaXPERT
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Chère consœur, cher confrère,
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| Retrouvez ci-après les dernières publications scientifiques issues de revues à comité de lecture dans votre domaine d'expertise. |
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| Publications du 06 septembre 2026 |
| Durability of efficacy, safety, and quality of life 5 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A: final phase 3 GENEr8-1 trial results |
| Research and Practice in Thrombosis and Haemostasis 2026; 10(3): 103416 |
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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| Advances in treatments for hemophilia A/B with inhibitors: current treatment gaps and promising therapies |
| Expert Review of Hematology 2026; 19(9): 1059-1076 |
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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| Long-term outcomes with emicizumab prophylaxis for haemophilia A in China: A multicentre, large-cohort retrospective study. |
| British Journal of Haematology (2026). |
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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| New and novel pharmacotherapies for hemophilia A: an update |
| Expert Opinion on Pharmacotherapy 2026; 27(13): 1467-1484 |
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...
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