Gene therapy for hemophilia clinical trials pipeline tool
Follow the progress of hemophilia gene therapy clinicals with the WFH Gene Therapy for Hemophilia Pipeline tool to track clinical trials and stay informed about current research. For more information about clinical trials, click here. The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. […]
WFH Educational: Treatment choice in an era of change | Virtual Summit 2020
In this session from the WFH 2020 Virtual Summit, Glenn Pierce, Johnny Mahlangu, David Page & Brian O’Mahony explore the patient and provider perceptions on treatment choices: how does each make a choice and process the risk-benefit analysis. The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment […]
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 […]
Substitution therapy | Virtual Summit 2020
Despite significant paradigm shifts in the medical management of hemophilia A with inhibitors and the expanding use of novel non-factor therapies, FVIII-based immune tolerance induction (ITI) is still the only effective means to inhibitor eradication. With the recently introduced Atlanta protocol, emicizumab prophylaxis is given in combination with 50-100 IU/kg FVIII three times a week. […]
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 […]
Practical Education on Bleeding Disorders: Knowledge for All | WFH webinar series | July 27 2021
Listen to this 2-hour webinar presented on July 27, 2021 to learn about global topics related to bleeding disorders. The session was moderated by Glenn Pierce (WFH Vice President, Medical) and Cedric Hermans (WFH Medical board member). Steven Pipe (Professor of Pediatrics and Pathology), Heiner Wedemeyer (Professor), Nathan Connell (Assistant Professor of Medicine), Dawn Rotellini […]
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 […]
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 […]