Emerging therapies for hemophilia: controversies and unanswered questions.
Ontology highlight
ABSTRACT:
SUBMITTER: Arruda VR
PROVIDER: S-EPMC5931262 | biostudies-literature | 2018
REPOSITORIES: biostudies-literature
Publications
Emerging therapies for hemophilia: controversies and unanswered questions.
Arruda Valder R VR Doshi Bhavya S BS Samelson-Jones Benjamin J BJ
F1000Research 20180424
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 thera ...[more]
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