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Publications on Friday July 24, 2026
Clinical Factors for Predicting Synovial Hypertrophy in Early Haemophilic Arthropathy on Prophylaxis: A Cross-Sectional Study of Patient- and Joint-Level Analyses Using Musculoskeletal Ultrasound
Haemophilia (2026).
Early identification of synovial hypertrophy is critical for preventing irreversible changes in haemophilic arthropathy (HA). Although prophylaxis has improved joint outcomes, it remains unclear which...
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Immunogenic implications of translational readthrough modulate the association of F8 nonsense mutations with inhibitors in Hemophilia A
Molecular Medicine (June 2026)
Among F8 mutation types, main determinants of inhibitor development after replacement therapy in Hemophilia A (HA), nonsense mutations display wide variation in the associated risk. Translational...
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INCREASED GENE THERAPY EFFICACY THROUGH THE USE OF EXTENDED HALF-LIFE CLOTTING FACTORS
Blood advances (July 2026)
of multiple clinical trials show significant interpatient variability in clinical outcomes, with a fraction of patients (up to 13%) even returning to prophylaxis. Another major challenge in current AAV...
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Effectiveness and Safety of Prophylactic Low-Dose Emicizumab in Young Children With Hemophilia A: A Multicenter Retrospective Study
eJHaem (2026).
Real-world data on low-dose emicizumab in young children with hemophilia A are limited. Methods: This multicenter retrospective study included 26 children (median age: 2.2 years) receiving individualized...
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