Patient Perspective: Hemophilia B and Gene Therapy

Rob Schroeder, a patient with hemophilia B describes his experience receiving gene therapy (etranacogene dezaparvovec). Hemophilia B is a genetic bleeding disorder due to dysfunction or deficiency of coagulation Factor IX. People with this condition may bleed for longer periods of time after injury or surgery. They are also susceptible to spontaneous bleeding in muscles, joints and organs, which can be extremely painful and, in some cases, life-threatening. As Mr. Schroeder explains, his chronic condition required him to be dependent on regular Factor IX infusions to manage his condition. Following his experience in a clinical trial and being given etranacogene dezaparvovec, he no longer required infusions with Factor IX and is relatively pain free. Mr. Schroeder also noted that the gene therapy has reduced his concerns about future health insurance issues that could arise if he was dependent on regular Factor IX infusions. To learn more about hemophilia B and other rare hematological disorders, visit checkrare.com/diseases/hematologic-disorders/