Hemophilia is a result of a mutation in one of the genes that usually gives instructions to make clotting factor proteins. This change -- or mutation -- prevents the clotting protein from working ...

Approval of valoctocogene roxaparvovec for adults with severe hemophilia A was based on data from the global phase III GENEr8-1 study, in which the therapy reduced the average annualized bleeding rate ...

Hemophilia B, also known as Christmas disease, is a bleeding disorder. Its characteristic feature is a deficiency of clotting factor IX. This is a protein present in the blood that helps with ...

Dutch firm UniQure presented results from an early study of AMT-060, a gene therapy it’s developing for patients with hemophilia B. To be clear, these are numbers from just the first handful of ...

SAN DIEGO — Promising early results from an ongoing randomized, open-label, single-arm, Phase 3 study could pave the way for a Pfizer product to become the second US Food and Drug Administration (FDA) ...