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 […]
The WFH Gene Therapy and World Bleeding Disorder Registries | Virtual Summit 2020
In this session from the WFH 2020 Virtual Summit, Barbara Konkle, Alfonso Iorio & Catherine Lambert will discuss & highlight two key WFH data programs: The WFH Gene Therapy Registry (GTR) and the WFH World Bleeding Disorders Registry (WBDR). With gene therapy for hemophilia on the horizon, there is an urgent need to develop a […]
Laboratory sciences: Laboratory issues and testing | Virtual Summit 2020
In this session from the WFH 2020 Virtual Summit, Steve Kitchen, Stefen Tiefenbacher, Anna Lowe and Annette Bowyer discuss: Gene therapy for FVIII and FIX: Will there be laboratory assay issues? Laboratory issues with isolated emicizumab therapy Laboratory issues when emicizumab and FVIII replacement are combined The WFH does not engage in the practice of […]
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 […]
Glenn Pierce and Steve Pipe discuss the latest updates on Gene Therapy for hemophilia | July 2021
The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH recommends that you contact your physician or local treatment centre. Before administering any products, the WFH urges patients to check dosages with a physician […]
Proceedings of the WFH’s Eleventh Global Forum on Research and Treatment Products for Bleeding Disorders
A summary of discussions held at the Eleventh WFH Global Forum on Research and Treatment Products for Bleeding Disorders which brings together patient groups, healthcare providers, researchers, regulators, industry representatives, and not-for-profit fractionators.
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 […]
Gene therapy: Is robust scientific inquiry the missing factor?
Listen to this 60-minute interactive webinar presented on 11 December 2020 to explore patient perspectives related to scientific inquiry of gene therapy. The session was moderated by David Page (Canadian Hemophilia Society). The panelists included Glenn Pierce, MD, PhD (WFH Vice President, Medical), Radoslaw Kaczmarek, PhD (Polish Hemophilia Society), Brian O’Mahony (Irish Haemophilia Society), and […]
The World Federation of Hemophilia Gene Therapy Registry – Informational Webinar
Listen to this 45-minute webinar presented on 9 November 2020 to learn more about the World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR). Barbara Konkle, MD (WFH Board of Directors), Glenn Pierce, MD, PhD (WFH VP Medical) and Wolfgang Miesbach, MD (GTR Steering Committee member) discuss the protocol and core data set, the implementation […]