What is gene therapy?

This patient handbook contains basic information about gene therapy for hemophilia. 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 […]

Medical sessions | WFH World Congress 2022

In these sessions from the WFH 2022 World Congress, David Lillicrap, Len Valentino, Sophie Susen, Mrinal Gounder, Rebecca Kruse-Jarres, Christine Mrakotsky, Roger Schutgens, Dan Hart, Margaret Rand, Karina Meijer Waander van Heerde, Keith Gomez, Emna Gouider, Charles Nakar, Brian R. Long, Tiago Lopes, Claire Pouplard, Radek Kaczmarek, Johnny Mahlangu, Steve Pipe, K. Rajender Reddy, Pratima […]

Questions to Ask Your Doctor Before Deciding to Take Gene Therapy

For people considering taking gene therapy, it is extremely important to learn as much as possible about if you are a good candidate, what the potential benefits and risks are, and what to expect during the procedure, and essential that your questions are answered. It may be helpful to write down a list of questions […]

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 […]

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 […]

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. […]