National Bleeding Disorders Foundation/X
Aug 31, 2025, 09:45
Slow Uptake of Hemophilia Gene Therapies: Challenges in Access and Adoption
National Bleeding Disorders Foundation (NBDF) shared on X:
”A new forum piece in Research and Practice in Thrombosis and Haemostasis explores the complex factors at play, from long-term efficacy and safety questions to cost, access, and patient decision-making.
Read the full open-access article.”
A recent RPTH forum article examines why the uptake of hemophilia gene therapy remains modest, citing concerns over long-term efficacy and safety, high costs, access barriers, and competition from effective non-factor therapies.
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