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Prolonging the Effects of Factor IX Replacement Therapy in Hemophilia B.

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Presentation on theme: "Prolonging the Effects of Factor IX Replacement Therapy in Hemophilia B."— Presentation transcript:

1 Prolonging the Effects of Factor IX Replacement Therapy in Hemophilia B

2

3 Introduction/Overview

4 Introduction/Overview (cont)

5 Importance of Prophylaxis, and Limitations on Its Application, in Hemophilia B

6 Key Unmet Need in Hemophilia B: Reducing Patient Burden of Disease

7 Improving Adherence in Hemophilia B

8 Potential Barriers to Adherence in Hemophilia Prophylaxis

9 Improving Adherence by Addressing the Barriers to Prophylaxis

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11 Surgical Experience With EHL rFIX Replacement Therapy

12 Annualized Bleeding Rates: Results From the B-LONG Study

13 Inhibitor Development in EHL Molecules for Treatment of Hemophilia B

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15 Annualized Bleeding Rates: Results From the Nonacog Beta Pegol Phase 3 Trial

16 The Importance of Maintaining Trough Levels to Prevent Breakthrough Bleeding

17 EHL Agents and Treatment Individualization

18 rFIX-FP Phase 3 Study in Hemophilia B: Study Design

19 rFIX-FP Phase 3 Study: AsBRs of Prophylaxis Patients

20 rFIX-FP Phase 3 Study: Safety Results

21 EHL Agents and Treatment Individualization

22 Phenotypic Variability Among PWH

23 Pharmacokinetic Characteristics of 3 EHL Agents

24 Evolution of FVIII/FIX Concentrates

25 Case Studies With Long-Acting FIX Therapies

26 Introduction/Overview

27 Case 1: 30-Year-Old Man Typically Treated on Demand

28 Case 2: Older Man With a Sedentary Lifestyle

29 Case 3: Patient Requiring Surgery

30 Case 4: Patient With a Frequent Bleeding Phenotype

31 Summary and Conclusions

32 Hemophilia B Treatment Goals

33 The Benefits of EHL Therapies in Hemophilia B

34 Transforming the Treatment Paradigm

35 Abbreviations

36 Abbreviations (cont)

37 Abbreviations (cont)


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