The fifth and final activity in the Clinical Advances in Gene Therapy for Hemophilia CME curriculum, Updates on Clinical Trials of Gene Therapy in Hemophilia, is a clinical article. Follow Thierry VandenDreissche (Brussels, Belgium) as he takes a deeper dive into the advances made during the recent early phase gene therapy clinical trials in hemophilia that have paved the way for the current pivotal phase 3 studies. The article examines the technical challenges faced by the initial approaches and the distinct solutions advanced for hemophilia A and B. It reviews the key results of the most promising studies to date and looks at the directions current research is taking for each. The questions that follow allow you to see how much you have learned about gene therapy clinical trials in hemophilia. Click the View Resource button to access the activity through the Medscape site. There you will have to register, which is free and straightforward.
This activity is part of the Clinical Advances in Gene Therapy for Hemophilia CME curriculum developed by the WFH in collaboration with the National Hemophilia Foundation (NHF), the European Haemophilia Consortium (EHC), and Medscape Education. These five online activities provide introductory information on gene therapy in hemophilia. While clinicians can qualify for CME credits all learners can obtain a completion certificate, or explore the activity to enhance their knowledge of gene therapy in hemophilia.
View the other activities in this curriculum:
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