Long-term safety and effectiveness tracking for a new hemophilia B treatment

This is a long-term observational study that tracks patients with hemophilia B who have received HEMGENIX, a new gene therapy treatment. The researchers will collect information about how well the medication works and any side effects patients experience over time. The study also follows a comparison group of hemophilia B patients on traditional preventive therapy to help doctors understand how the new treatment compares.

Hemophilia B is a rare bleeding disorder, and this medication is a relatively new option that works very differently from traditional treatments. Doctors need real-world information about whether this treatment keeps working safely over many years, since it was only recently approved for patients.