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Rare Blood Disorders

Explore facts and recommendations for pharmacological treatments for rare blood disorders.

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rare blood disorders Video

Concizumab Mechanism of Action

This video discusses the mechanism of action of an investigational medicine, concizumab.

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rare blood disorders PDF

Risk Mitigation During Concizumab Prophylaxis in Phase 3 Clinical Trials in Patients With Hemophilia A/B With and Without Inhibitors

This poster reviews the risk mitigation strategy and breakthrough bleed management guidance for patients treated with concizumab in clinical trials.

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Improved Quality of Life and Satisfaction With N8-GP in US Patients

This poster reviews the health-related quality of life and satisfaction in US patients with severe hemophilia A receiving N8-GP in the pathfinder trials.

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Longitudinal Efficacy and Safety of N9-GP for Prophylaxis and Bleed Control in US Patients

This poster reviews the efficacy and safety of N9-GP in US patients on prophylactic and on-demand therapy.

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Emerging Non-Factor Therapies for Hemophilia

Mechanism of action for emerging therapies to treat hemophilia without using replacement coagulation factor.

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