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Mid-Year Hemophilia Update

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Presentation on theme: "Mid-Year Hemophilia Update"— Presentation transcript:

1 Mid-Year Hemophilia Update

2

3 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.

4 Extended Half-Life Factor Products

5 Extended Half-Life Factor Products (cont)

6 Inhibitors

7 Initiating Factor Product

8 ITI

9 ITI (cont)

10 Monitoring and PK Studies

11 Population PK Studies

12 Accuracy of Lab Assays

13 Challenges of Assays

14 Recent Data on Assays at ISTH

15 Surgery

16 Surgery Trials at ISTH

17 Emicizumab

18 Emicizumab (cont)

19 Emicizumab: Safety

20 Fitusiran

21 Cautions

22 Coagulation Cascade

23 Other Novel Approaches

24 Further Thoughts on Novel Products

25 Gene Therapy

26 The Future

27 Abbreviations

28 Abbreviations (cont)


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