Rare Disease - Episode 4
Hemophilia is a congenital deficiency of vital clotting factors that are needed to form blood clots at the site of vascular injury. As an X-linked recessive disease, only males are clinically impacted by hemophilia, whereas females are only carriers for the bleeding disorder. Often presenting in early childhood, patients who are not optimally treated may experience bleeding episodes, reduced physical activity, decreased social participation, and long-term hemophilic arthropathy. As a consequence, hemophilia is a serious condition that is tied to substantial clinical and economic burdens, a negative impact on patient quality of life, and must be managed appropriately.