In this session from the WFH 2020 Virtual Summit, Radoslaw Kaczmarek, Flora Peyvandi, Dan Hart & Johnny Mahlangu discuss novel products newly arrived on the market, that are very different from traditional clotting factor concentrates and pose new questions about safety and clinical use. The WFH does not engage in the practice of medicine and […]
In this session from the WFH 2020 World Summit, Glenn Pierce will examine the evolution of gene therapy while addressing the unknowns that need to be answered. Hemophilia is at the dawn of a new era in therapeutic management, one that can generate greater protection from bleeding and a functional cure in some individuals. The […]
With regulatory approval of the first gene therapy product expected, there is an urgent need for a mechanism to collect long-term data on safety and variability and durability of efficacy. There will be elements required by regulators for post marketing surveillance and additional data needed to enhance our understanding of gene therapy outcomes and their […]
We are still learning about gene therapy. Each patient experience is different, and gene therapy is not for everyone – either by choice or by exclusion because of the antibodies. In this session from the WFH 2020 Virtual Summit, Debra Pollard, John Pasi, David Page, Garrett Hayes, Rob Schroeder and Enrique Preza Hernandez discuss: What […]
In the Tuesday afternoon session entitled Prophylaxis: Global Models of Low-Dose Prophylaxis, Shashikant Apte presents the results of the implementation of tertiary prophylaxis programs in India, focusing on reduction of annual bleed rate (ABR) in hemophilia A and B.
In the Sunday afternoon session entitled Gene Therapy: Back to Basics, Glenn Pierce explains the state of the art and the future prospects of gene therapy, gene editing, and cell therapy, in the context of hemophilia.