WFH Gene Therapy Registry – User guide for people with hemophilia
This document is an introductory guide for people with hemophilia who wish to join the World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR). It answers basic questions about the registry, its purpose, and what participants can expect. Please reach out to WFH with questions regarding participation in the registry at [email protected] WFH Gene Therapy […]
WFH Gene Therapy Registry – User guide for hemophilia treatment centres
The World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR) welcomes the participation of all Hemophilia Treatment Centres (HTCs) involved in gene therapy. This includes HTCs that administer gene therapy and those that manage or follow-up with patients who have received gene therapy at other centres. This user guide will orient HTCs to the WFH […]
WFH Gene Therapy Registry – Basics of hemophilia, gene therapy, and long-term data collection
This document is an introductory guide to the World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR). It answers basic questions about the registry, its purpose, and what participants can expect. The guide is designed to help those who receive gene therapy for hemophilia and their care teams to decide if they should participate in […]
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 […]