Barriers to gene therapy, understanding the concerns people with haemophilia have: an exigency sub-study
Abstract 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 inhibitor...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
| Published: |
BMC
2024-02-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s13023-024-03068-2 |