Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study.

ABSTRACT:

Background

Gene therapy has the potential to offer people with haemophilia (PwH) a life free from bleeding and the burden posed by current treatment regimens. To date, gene therapy has only been available in clinical trial settings, to PwH without pre-existing or historical factor inhibitors, significant concomitant liver damage or pre-existing neutralising antibodies to the adeno-associated viruses used to deliver the therapy. Thus, most PwH treated at centres not currently involved in gene therapy trials, either as a referral/follow-up centre or as a dosing centre, have been unable to access the therapy. This Exigency sub-study aims to gain a greater understanding of the opinions of PwH in the United Kingdom who have not had access to gene therapy: asking what they understand, wh

SUBMITTER: Fletcher S 

PROVIDER: S-EPMC10859013 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature