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Mid-Year Hemophilia Update
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This program will include a discussion of off-label treatment and investigational agents not approved by the FDA for use in the US, and data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.
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Extended Half-Life Factor Products
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Extended Half-Life Factor Products (cont)
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Inhibitors
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Initiating Factor Product
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ITI
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ITI (cont)
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Monitoring and PK Studies
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Population PK Studies
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Accuracy of Lab Assays
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Challenges of Assays
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Recent Data on Assays at ISTH
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Surgery
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Surgery Trials at ISTH
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Emicizumab
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Emicizumab (cont)
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Emicizumab: Safety
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Fitusiran
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Cautions
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Coagulation Cascade
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Other Novel Approaches
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Further Thoughts on Novel Products
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Gene Therapy
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The Future
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Abbreviations
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Abbreviations (cont)
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