Better data, better patient outcomes: 20 years of data collection | Virtual Summit 2020
This workshop from the WFH 2020 Virtual Summit, presented by Ellia Tootoonchian, Mark Skinner, Donna Coffin, Alfonso Iorio, Emna Gouider-Belhadjali and Jeff Stonebraker, is intended for healthcare professionals engaged in clinical research and patient leaders and organizations who have an active interest in using health data in their work. The WFH does not engage in […]
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 […]
Medical Plenary: Gene Therapy | Virtual Summit 2020
In this session from the WFH 2020 Virtual Summit, Steve Pipe and Luigi Naldini explore the promise of gene therapy and the broad-based multi-stakeholder preparation that is needed to deliver on this achievement. Steve Pipe explores that, although we currently stand on the threshold of this achievement, delivering on this promise will require broad-based multi-stakeholder […]
Inhibitors – Think differently | Virtual Summit 2020
The development of inhibitory antibodies to therapeutic factor VIII (FVIII) in up to 40% of people with severe hemophilia A (HA) is the major complication in treatment/prevention of hemorrhages. The reasons some people with HA develop inhibitors while others do not, remain unclear. In this session from the WFH 2020 Virtual Summit, Sebastien Lacroix-Desmazes, Kathleen […]