After several decades of very limited or no treatment at all, the haemophilia patients who have the added burden of inhibitors, are facing an era where new possibilities of treatment become available also for them. 

In this brief article we would like to offer  an insight into the gene therapy, a treatment option that is currently not available for people with inhibitors, but worth looking into nevertheless.  For gene therapy prospects for people with inhibitors you can read in our Ask the Expert Section (available after 1st June) or listen during the live discussion with the EHC President Declan Noone on 25th May

The idea of gene therapy for haemophilia is using a modified virus (so that it is unable to cause a disease) to introduce a copy of the gene into the patients body that then encodes the clotting factor missing. Following treatment with the virus, patients should begin producing their own clotting factor normally.

To inform the patient community about the gene therapy, the EHC has created a series of videos that focus on gene therapy and haemophilia from both patient and expert points of view. The five-part series has been created to help patients and the general public understand and demystify some of the concepts related to gene therapy in haemophilia.

This series is the first major piece of patient-focused discussion and education about gene therapy and haemophilia.

What is gene therapy? with Prof. Flora Peyvandi

An overview in gene therapy for haemophilia A and B with Dr. Paul Giangrande

How does a clinical trial in gene therapy for haemophilia work? with Dr. Dan Hart

Safety and gene therapy with Prof. Michael Makris

Gene therapy: A patient’s perspective with Jack Grehan


Furthermore, in anticipation of broader use of gene therapies in bleeding disorders, the EHC in collaboration with European Association of Haemophilia and Allied Disorders (EAHAD) have developed a EAHAD-EHC Joint Statement on: Promoting hub-and-spoke model for the treatment of haemophilia and rare bleeding disorders using gene therapies.




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