8.00 Registration & Networking Coffee

8:50 am Chair’s Opening Remarks

The Approach to Patients with Bleeding Disorders

9:00 am A Patient-Centric Approach to Drug Development Across the Lifecycle

  • Eileen Sawyer Vice President, Global Medical Affairs, uniQure, Inc

Synopsis

• Incorporating patient perspectives and how to involve patients in gene therapy drug development
• At what stage of the program lifecycle should patients be involved in order to meet their needs and to optimize trial conduct
• How to provide a compelling value proposition for multiple stakeholders

9:30 am Fitusiran and Mechanisms of Enhanced Thrombin Generation

Synopsis

• Elucidate how fitusiran modulates the coagulation cascade via inhibition of antithrombin
• Discussing how this leads to enhanced levels of thrombin generation

10:00 am Sponsored Talk by Machaon Diagnostics

10.30 Morning Refreshments & Networking

Addressing the Unmet Clinical Need in the Pediatric Population

11:30 am Discussing the Key Challenges Associated with Treating Pediatric Patients with Hemophilia

Synopsis

• How can administrative procedures be improved to reduce the trauma children have to go through when receiving regular treatments?
• What is the potential of gene therapy treating children when there is a high turnover of cells in the liver?
• Discussing the ethical issues parents face when choosing a treatment plan for their child

Understanding the Impact on Women with Hemophilia

12:00 pm Challenges Facing Providers and Women with Hemophilia

Synopsis

• To describe the evolving nomenclature
• To Characterize the challenges in current diagnostic testing and discrepancy in levels and clinical severity
• To provide an overview of evolving management strategies

12:30 pm Sponsored talk by American Thrombosis & Hemostasis Network

13.00 Lunch

Treating Von Willebrand Disease

2:00 pm Determining Effective Diagnostics and Treatment for Patients with Von Willebrand Disease

  • Jonathan Roberts Associate Director - Medical & Research & Assistant Professor of Pediatrics, Bleeding & Clotting Disorders Institute

Synopsis

• Improving testing methods to identify phenotypic variants of VBW
• Could gene therapy provide a long lasting alternative to patients?
• What novel drugs are being developed to improve patient care?

Determining Effective Treatments for the Inhibitor population

2:30 pm Current Treatment Options Available for this Unique Subset of Patients

15.00 Afternoon Refreshments & Scientific Poster Session

Novel Drugs for the Treatment of Acquired Hemophilia

4:00 pm Specific Challenges Associated with Patients with Acquired Hemophilia

  • Miguel Escobar Professor of Medicine and Pediatrics, University of Texas Health Science Center at Houston

Synopsis

• Discussing the unmet needs for patients with acquired hemophilia
• Assessing the current available drugs and if they meet the need of the patients
• What other therapies could be used to treat these patients

4:30 pm Chair’s Closing Remarks & End of Day One

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