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Rare Diseases: Hematology

We are relentless in our pursuit of a world without bleeds. Beyond delivering new medicines, we are dedicated in our efforts to encourage early diagnosis and propel patients towards optimal personalized care, and help them remain on the right therapy with the goal of achieving the best possible long-term outcomes.

We are investigating important scientific platforms, such as antibody and gene therapy, so that we are best positioned to discover innovative therapies tomorrow—while ensuring patients are cared for with use of our broad portfolio today.

We’re committed to caring for patients with bleeding disorders including:


Hemophilia is an inherited bleeding disorder that mostly affects males. People with hemophilia lack or have low levels of certain proteins called clotting factors, so their blood doesn’t clot properly. Over 400,000 people worldwide are living with hemophilia.

Von Willebrand Disease

Von Willebrand Disease (VWD), the most common hereditary bleeding disorder, is caused by deficiency or dysfunction of the von Willebrand factor (VWF) where a solid clot can’t form or takes longer to form, resulting in prolonged bleeding.1 VWD occurs in approximately 1% of the world’s population, including as many as 3.2 million people in the U.S. alone.2 9 out of 10 people with VWD have not been diagnosed.


Canadian Hemophilia Society. All about von Willebrand disease…for people with von Willebrand disease and their families. 3rd ed. http://www.hemophilia.ca/files/All%20About%20VWD%202011%20-%20FINAL.pdf Published December 2011. Accessed April 17, 2017

Centers for Disease Control and Prevention Website. https://www.cdc.gov/ncbddd/vwd/facts.html Accessed January 23, 2018