It was found that those with severe hemophilia B have less than 1% of normal factor IX activity, often experiencing spontaneous bleeding that can cause joint damage or life-threatening events.

The results of gene therapy for hemophilia B contrast with those of factor replacement therapies, where lifelong infusions ...

Gene therapy has proved to be a safe long-term treatment for haemophilia B. The benefits of a single dose persist for at ...

With the approval of the FDA, garadacimab can help patients with hereditary angioedema (HAE) prevent attacks by shutting down the contact pathway.