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Advancements and Approaches in Hemophilia Treatment
Hemophilia is a rare, inherited bleeding disorder where the blood does not clot properly due to a deficiency or absence of clotting factors. This condition, primarily affecting males, can lead to prolonged bleeding after injuries, spontaneous internal bleeding, and joint damage over time. While there is no universal cure, significant progress in treatment options has transformed the quality of life for individuals living with hemophilia.
The cornerstone of hemophilia treatment has historically been replacement therapy, where the missing clotting factors are administered intravenously. This therapy can be delivered on-demand, during bleeding episodes, or as a prophylactic measure to prevent spontaneous bleeding. Prophylactic treatment has been particularly transformative for children with hemophilia, reducing joint damage and improving overall mobility. Recent improvements in factor concentrates, such as recombinant products, have reduced the risk of infections previously associated with plasma-derived therapies.
In addition to conventional factor replacement,…