A recent study on hemophilia B led by Rasko et al.
(2025) investigated gene therapy using fidanacogene elaparvovec to sustain clotting factor IX activity, which is crucial for blood clotting in hemophilia B patients.
The study demonstrated that this therapy maintained factor IX activity within the mild hemophilia range over several years of follow-up.
These findings suggest gene therapy could offer long-term management of hemophilia B by stabilizing clotting factor levels, potentially reducing bleeding episodes and improving patients' quality of life.
The sustained factor IX activity indicates gene therapy may provide a more consistent treatment compared to frequent clotting factor infusions, offering hope for more effective and lasting treatment options.
Clinical Trials
This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks:
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