Article

Emerging therapies for hemophilia: controversies and unanswered questions

2018-04-24

Abstract excerpt

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...

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Literature Corpus work: a7bc7131-4313-5e8f-836f-9dadeb3552d8
DOI: 10.12688/f1000research.12491.1

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