Gene therapy for hemophilia B allowed almost three-fourths of patients to discontinue prophylactic factor IX therapy with no ...

Avigen is investigating the use of AAV vectors to deliver the factor IX gene for the treatment of hemophilia B and the factor VIII gene for hemophilia A. Preclinical efficacy and safety studies in ...

The frequency of mutations in the factor VIII or IX gene is consistent. However, it turns out that anyone can be affected by hemophilia. Hemophilia could potentially develop in every ten ...