“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

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 th...

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Bibliographic Details
Main Authors: Khair Kate, Nagra Ranjit, Gorman Richard, Fletcher Simon, Jenner Kathryn, Trim Joe, Collings Shuk-Li
Format: Article
Language:English
Published: Sciendo 2025-03-01
Series:The Journal of Haemophilia Practice
Online Access:https://doi.org/10.2478/jhp-2025-0001
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