Yesterday’s pre-Summit session, “Uncertainty in an Era of Transformative Therapy for Hemophilia: Addressing the Unknowns” covered many aspects related to the potential for gene therapy to provide a cure for many bleeding disorders. The talk was given by Glenn Pierce, MD, PhD, WFH Vice-President, Medical.
Since the 1960s, the pace of innovation in hemophilia treatment has been fast and furious and occasionally with unintended consequences. As newer technologies are harnessed to better treat, and potentially cure, hemophilia A and B, an understanding of their underlying scientific principles and their benefits and risks are essential for all stakeholders. The session looked at the use of gene transfer as an effective long-term treatment for hemophilia, and addressed the unknowns of gene therapy, that still need to be answered.
To watch sessions from the WFH Virtual Summit – Connecting the Global Bleeding Disorders Community, please click here.