Gene Therapy Working Group

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The EAHAD Gene Therapy Working Group was formed in 2019.


The working group will strive to be at the forefront of providing critical information to physicians and patients about gene therapy in terms of practicability and safety.


Its goals 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.


The members of the working group are:

Prof. Dr Wolfgang Miesbach
HaematologistGoethe University HospitalGermany
Prof. Ana Boban HaematologistUniversity Hospital Center ZagrebCroatia
Dr Michiel CoppensHaematologistAmsterdam UMCThe Netherlands
Dr Robert KlamrothHaematologistVivantes Klinikum im FriedrichshainGermany
Prof. Michael MakrisHaematologistUniversity of SheffieldUnited Kingdom
Greta MuldersNurseErasmus MCThe Netherlands
Declan NoonePresidentEuropean Haemophilia ConsortiumBelgium
Prof. Flora PeyvandiHaematologistUniversity of MilanItaly
Dr Víctor Jiménez YusteHaematologistLa Paz University HospitalSpain


EAHAD Gene Therapy Working Group activities

Delivery of AAV-based gene therapy through haemophilia centres—A need for re-evaluation of infrastructure and comprehensive care: A Joint publication of EAHAD and EHC
Selection of EAHAD 2021 Gene Therapy highlights (available on the ISTH GT website)
Gene Therapy Key Components of Clinical Care Session (available on the ISTH GT website)