Emerging therapies for hemophilia: controversies and unanswered questions

F1000Research
Valder R ArrudaBenjamin J Samelson-Jones

Abstract

Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change hemophilia care dramatically, several challenges and questions remain regarding broader applicability, long-term safety, and which option to pursue for each patient. Here, we review these emerging therapies with a focus on controversies and unanswered questions in each category.

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Jun 11, 2019·Clinical and Applied Thrombosis/hemostasis : Official Journal of the International Academy of Clinical and Applied Thrombosis/Hemostasis·Aveen M Raouf AbdulqaderSarwar Noori Mahmood
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Clinical Trials Mentioned

NCT03093480

Software Mentioned

uniQure
SIPPET

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