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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

The Role of PKR Activation in Sickle Cell Disease

This video shares the role of PKR activation in Sickle Cell Disease.

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Rare Blood Disorders PDF

Sickle Cell Disease Clinical Development Programs

Information about various clinical trials (ASCENT1, HIBISCUS, GLADIOLUS, etc) to evaluate safety and efficacy for sickle cell disease and/or other hemoglobinopathies.

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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

Emerging Non-Factor Therapies for Hemophilia

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

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Continuing Education

Find accredited opportunities in rare blood disorders education.

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