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This analysis reveals challenges in adopting gene therapy in haemophilia, suggesting improved outcomes through data collection and monitoring.
Despite its transformative potential, slow real-world adoption is observed, constrained by scientific, operational, and reimbursement challenges. Ongoing data collection via registries and harmonized monitoring pathways will be vital for optimizing outcomes and delineating long-term safety profiles for gene therapy in haemophilia clinical practice.
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Miesbach et al. (2026) studied this question.
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