Abstract

In addition to the objective clinical and biological results of recent clinical trials, the experiences of patients treated with gene therapy should be surveyed and shared with the community. In this original study, 16 patients (11 HA and 5 HB) who took part in clinical trials from the UK, 10 accompanied by a family member, provide valuable information of their experience of gene therapy [12]. Beyond biological (FVIII or FIX levels after gene therapy) and clinical (number of bleeds) results, it is important to be able to share with potential future candidates high-quality real-life data reflecting the personal experiences and perceptions of patients already treated.

Details

Title
Haemophilia gene therapy: experiences and lessons from treated patients
Author
Hermans, Cedric  VIAFID ORCID Logo 
Pages
1-3
Section
Editorial
Publication year
2022
Publication date
2022
Publisher
BioMed Central
e-ISSN
17501172
Source type
Scholarly Journal
Language of publication
English
DOI
https://doi.org/10.1186/s13023-022-02313-w
ProQuest document ID
2652165023
Copyright
© 2022. This work is licensed under http://creativecommons.org/licenses/by/4.0/ (the “License”). Notwithstanding the ProQuest Terms and Conditions, you may use this content in accordance with the terms of the License.