Patient Perspectives | Sanofi Rare Blood Disorders

Hemophilia A and B

The most common form of hemophilia is hemophilia A, which is caused by a deficiency in factor VIII. Hemophilia B is caused by a deficiency in factor IX. Both factor VIII and factor IX are proteins that play an important role in forming blood clots that prevent internal bleeding or help stop bleeding after external injuries such as cuts.

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Acquired thrombotic thrombocytopenic purpura

Acquired thrombotic thrombocytopenic purpura (aTTP) is a rare, life-threatening, autoimmune blood clotting disorder that affects about 2,000 people in the US every year.

The disease is caused by a deficiency of an enzyme called ADAMTS13. The lack of functioning ADAMTS13 enzyme results in blood platelets sticking together and forming small clots (microthrombi) throughout the body, which can lead to neurological, cardiac, renal and gastrointestinal damage, and, in some cases, death.

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