The evolving landscape of gene therapy for congenital haemophilia: An unprecedented, problematic but promising opportunity for worldwide clinical studies.

Abstract excerpt

With liver-directed gene therapy, congenital haemophilia has the potential to progress from an incurable to a phenotypically curable condition. However, the proportion of haemophilia population likely to benefit from gene therapy remains to be established. Achieving a phenotypic curative goal is presently hampered by: 1) availability of effective treatments (e.g. extended half-life products, non-factor therapies)...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Cost-Benefit Analysis
• Share to Disease Management