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

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, signif...

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Bibliographic Details
Published in:	Orphanet journal of rare diseases Vol. 19; no. 1; pp. 59 - 12
Main Authors:	Fletcher, Simon, Jenner, Kathryn, Holland, Michael, Khair, Kate
Format:	Journal Article
Language:	English
Published:	London BioMed Central 10.02.2024 BioMed Central Ltd Springer Nature B.V BMC
Subjects:	Beliefs, opinions and attitudes Bureaucracy Care and treatment Clinical trials Data collection Factor IX deficiency Gene therapy Haemophilia Health care disparities Hemophilia Hemophiliacs Human Genetics Medicine Medicine & Public Health Outcomes Pharmacology/Toxicology Quality of life Shared decision making Social aspects United Kingdom United Kingdom > UK Haemophilia Gene therapy Outcomes Shared decision-making Quality of life
ISSN:	1750-1172, 1750-1172
Online Access:	Get full text
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