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Hemophilia is a blood disorder caused by a shortage of specific clotting factors, leading to bleeding issues. While there is no cure, treatments aim to manage symptoms and improve quality of life. Primary treatments include clotting factor replacement therapy, bypassing agents, gene therapies, and nonfactor replacement therapies. Experimental treatments like CRISPR/Cas9 are also being explored.
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Hemophilia is classified as mild, moderate, or severe, depending on the level of clotting factor in the blood. About 70% of those with hemophilia have the severe form. The most important life-threatening complications of the disease are bleeding in the skull and hemorrhages in soft tissue around airways or other…