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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Vydané v:	Orphanet journal of rare diseases Ročník 19; číslo 1; s. 59 - 12
Hlavní autori:	Fletcher, Simon, Jenner, Kathryn, Holland, Michael, Khair, Kate
Médium:	Journal Article
Jazyk:	English
Vydavateľské údaje:	London BioMed Central 10.02.2024 BioMed Central Ltd Springer Nature B.V BMC
Predmet:	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
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