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 impact on the lives of people with hemophilia.
In this session from the WFH 2020 Virtual Summit, Barbara Konkle, Michael Recht, Caroline Voltz and Peter Marks discuss efforts underway by the American Thrombosis and Hemostasis Network and the World Federation of Hemophilia to collect long-term harmonized data and considerations of the European and US regulatory agencies, which will inform ongoing data collection.
For people considering taking gene therapy, it is extremely important to learn as much as possible about if you are
This document is an introductory guide for people with hemophilia who wish to join the World Federation of Hemophilia (WFH)
The World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR) welcomes the participation of all Hemophilia Treatment Centres (HTCs) involved
This document is an introductory guide to the World Federation of Hemophilia (WFH) Gene Therapy Registry (GTR). It answers basic