How do you approach deciding which patients with hemophilia B to recommend for gene therapy?

There is no standard protocol for who gene therapy is recommended for, but certainly, potential candidates must meet all criteria according to the manufacturer's recommendations. Gray areas may exist if there is some preexisting liver disease, where hepatology consultation is desired to help determi...

Those of us involved in the care of patients with hemophilia feel like” kids in a candy store” with all these new wonderful treatments, as just in the last year we’ve had 3 FDA approvals of rebalancing agents-fitusiran, concizumab, and marstacimab, and the FIX gene therapy product Hemgenix has been ...

As mentioned above, there is no standard protocol. However, an example criterion used by one center to help decide eligibility for HEMGENIX is listed below. These guidelines were based on the clinical criteria used in the HOPE-B trial and the FDA package insert for HEMGENIX.

Consider HEMGENIX for ad...