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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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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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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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 low-dose emicizumab (2019-2025)....
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