Journal article
“I’d go on a hike with my kids… that would make me feel so happy.” Recognising what people with haemophilia B identify as meaningful when considering personal goals and the potential for gene therapy
- Abstract:
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Introduction: Historically people with haemophilia B (PwHB) have experienced adverse clinical outcomes including joint bleeds, pain and arthropathy. The current management of severe and moderate haemophilia B in the UK is largely through the use of factor replacement therapy often given as prophylaxis. Despite this, research reveals reduced quality of life (QoL) and psychological burden in PwHB of all severities, highlighting unmet need. Contemporary therapeutic developments including extende...
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- Publication status:
- Published
- Peer review status:
- Peer reviewed
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- Files:
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(Preview, Version of Record, Version of record, pdf, 1.4MB, Terms of use)
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- Publisher copy:
- 10.2478/jhp-2025-0001
Authors
- Publisher:
- Paradigm Publishing
- Journal:
- The Journal of Haemophilia Practice More from this journal
- Volume:
- 12
- Issue:
- 1
- Pages:
- 1-14
- Publication date:
- 2025-03-19
- DOI:
- EISSN:
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2055-3390
- ISSN:
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2055-3390
- Language:
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English
- Keywords:
- Pubs id:
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2301411
- Local pid:
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pubs:2301411
- Source identifiers:
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2785108
- Deposit date:
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2025-03-20
- ARK identifier:
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- Copyright date:
- 2025
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