The Exigency Study | Exploring experiences of gene therapy in haemophilia
Clinical trials for haemophilia gene therapy have been going on since the early 1990s. With
the EMA recently granting conditional marketing approval (CMA) of the gene therapy for haemophilia A, and now B, it may soon become a treatment option for some people with severe haemophilia.
Haemnet’s Exigency study has been exploring the knowledge, expectations and experiences of gene therapy among people in the UK haemophilia community. As well as speaking with people who have had haemophilia gene therapy as part of a clinical trial, the research team spoke with people who were unable to have gene therapy in a trial, people who don’t want to have gene therapy, family members, parents of children with haemophilia and health care professionals. Some of the study findings are presented in this short animation.
Further reading
Khair K, Steadman L, Chaplin S, Holland M, Jenner K, Fletcher S. Parental perspectives on gene therapy for children with haemophilia: The Exigency study. Haemophilia 2021; 27(1):120-128. doi: 10.1111/hae.14188
Fletcher S, Jenner K, Holland M, Chaplin S, Khair K. An exploration of why men with severe haemophilia might not want gene therapy: The exigency study. Haemophilia 2021; 27:11-15. doi: 10.1111/hae.14378
Fletcher S, Jenner K, Pembroke L, Holland M, Khair K. The experiences of people with haemophilia, and their families, of gene therapy in a clinical trial setting: Regaining Control, the Exigency Study. Orphanet Journal of Rare Diseases. 2022; 17:155. doi: 10.1186/s13023-022-02256-2
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