What are possible future treatments for hemophilia?

What are possible future treatments for hemophilia?

Multiple trials and studies are underway to examine the possibility to use gene therapy to replace the defective genes in hemophilia. To date, stable and sustained production of the deficient clotting factors has not been achieved in humans, but this is an area of active investigation that holds great promise for the future.

Hemophilia At A Glance

• Hemophilia is one of a group of inherited bleeding disorders that cause abnormal or exaggerated bleeding and poor blood clotting.
• Hemophilia A and B are inherited in an X-linked recessive genetic pattern, so males are commonly affected while females are usually carriers of the disease.
• Hemophilia A is caused by a deficiency of clotting Factor VIII, while hemophilia B (also called Christmas disease) results from a deficiency of Factor IX.
• Hemophilia varies in its severity among affected individuals.
• Symptoms include excessive bleeding from any site in the body; long-term damage to joints from repeated bleeding episodes is characteristic.
• Treatment involves coagulation factor replacement therapy.
• The formation of inhibitors to the treatment factor concentrates is a significant complication of treatment.
• Gene therapy treatments are a source of active research and hold promise for the future.