Mid-Year Hemophilia Update

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Presentation transcript:

Mid-Year Hemophilia Update

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.

Extended Half-Life Factor Products

Extended Half-Life Factor Products (cont)

Inhibitors

Initiating Factor Product

ITI

ITI (cont)

Monitoring and PK Studies

Population PK Studies

Accuracy of Lab Assays

Challenges of Assays

Recent Data on Assays at ISTH

Surgery

Surgery Trials at ISTH

Emicizumab

Emicizumab (cont)

Emicizumab: Safety

Fitusiran

Cautions

Coagulation Cascade

Other Novel Approaches

Further Thoughts on Novel Products

Gene Therapy

The Future

Abbreviations

Abbreviations (cont)