Treatments for bleeding disorders: Current environment & pipeline products | Virtual Summit 2020
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 […]
Uncertainty in an era of transformative therapy for hemophilia | Virtual Summit 2020
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 […]
Critical need for harmonized post marketing surveillance in gene therapy | Virtual Summit 2020
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 […]
Gene therapy: the unfolding story | Virtual Summit 2020
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 […]
Low-Dose Prophylaxis – India Experience
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.
Gene Therapy for Haemophilia: What, Why, How, When, Who, and Where?
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.