Rare Blood Disorders
Explore facts and recommendations for pharmacological treatments for rare blood disorders.
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The Role of PKR Activation in Sickle Cell Disease
This video shares the role of PKR activation in Sickle Cell Disease.
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.
Concizumab Mechanism of Action
This video discusses the mechanism of action of an investigational medicine, concizumab.
Emerging Non-Factor Therapies for Hemophilia
Mechanism of action for emerging therapies to treat hemophilia without using replacement coagulation factor.