EAHAD is pleased to announce the formation of a Working Group on Gene Therapy, following the approval of a proposal to the Executive Committee in September. Chaired by Prof. Dr Wolfgang Miesbach, the Working Group will have a mandate of two years and will be comprised of six to eight members. A patient representative will also be invited to take part.
The tentative goals of the Working Group are:
– To investigate gene therapy safety and its outcome parameters.
– To provide updates on recent developments in terms of alanine aminotransferase (ALT) results and safety outcomes.
– To collect data on the different gene therapy trials, tests, and adverse events.
– To inform patients and medical professionals.
With gene therapy having the potential to create a paradigm shift in haemophilia as we enter a new decade, the EAHAD Gene Therapy working group will strive to be at the forefront of providing critical information to physicians and patients about this new treatment option in terms of practicability and safety.