Therapeutic Focus

Glanzmann thrombasthenia (GT) is a congenital severe, lifelong bleeding disorder characterized by deficient or dysfunctional glycoprotein IIb/IIIa expression on platelets, which impairs platelet-fibrinogen binding and platelet aggregation during primary hemostasis. Patients suffer from what can be a devastating combination of life-threatening acute hemorrhages and relentless chronic bleeding that profoundly impact patients’ physical health, mental health, and quality of life.

Von Willebrand Disease (VWD) is the most common inherited bleeding disorder. It is characterized by both life-threatening hemorrhagic events and relentless mucocutaneous bleeding episodes that combine to inflict significant harm to physical health, mental well-being, and quality of life. Women face a particularly severe burden from heavy menstrual bleeding, which can profoundly impact quality of life, reproductive choices, and iron status. Even patients with Type 1 VWD, which has traditionally been considered “mild”, experience a serious clinical burden. VWD results from reduced quantity or function of von Willebrand factor (VWF), a plasma protein essential for normal hemostasis through its dual role in platelet adhesion and as a carrier protein protecting coagulation Factor VIII from degradation.

Factor VII deficiency is a congenital severe bleeding disorder characterized by reduced levels of Factor VII, a naturally circulating blood coagulation protein. Patients with clinically severe Factor VII deficiency suffer from recurrent, unpredictable, life-threatening or potentially disabling bleeding at critical sites, such as in the central nervous system, gastrointestinal tract and intra-articular locations, as well as recurrent mucocutaneous bleeds of the nose and gums with additional risks for female patients, consisting of heavy menstrual bleeding and potentially life-threatening post-partum hemorrhage.