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Disease: Haemophilia

Last update: July 2009

Intro

Haemophilia is an inherited disorder where the blood does not clot properly. Only men are affected. Once a crippling or even fatal condition, the pharmaceutical industry has introduced preparations to control bleeding and allow many patients to lead near-normal lives.

FAQ
  • What is it?

    Haemophilia is an inherited disorder of blood clotting. The lack of blood clotting factors (notably Factor VIII and Factor IX) means that bleeding is prolonged. This causes bruising and bleeding into the joints. If the bleeding cannot be controlled, crippling deformities can result.
      

  • Who gets it?

    Haemophilia affects men almost exclusively. The genetic deficiency is passed on by women who usually have no bleeding problems. There are more than 22,000 European patients with haemophilia.
      

  • What can be done about it?

    There is currently no cure for haemophilia. However, the efforts of the pharmaceutical industry have resulted in preparations containing Factor VIII and Factor IX from blood donations or genetic engeneering. These make it possible even for people with severe forms of the disease to control bleeding and live near-normal lives.
      

  • What does the future hold?

    One field of research is devoted to finding ways of producing Factor VIII and Factor IX from sources other than blood donations. Another is to use gene therapy to allow a person with haemophilia to produce their own clotting factors. Advances in this area are exciting because this would represent a cure for this disease.