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Although hemophilia has no cure, approved treatments like factor replacement therapies, bypassing agents, and gene therapies help manage symptoms. Experimental treatments, including CRISPR/Cas9 gene editing and Pfizer's giroctocogene fitelparvovec, are also being developed to provide long-term solutions for hemophilia A.
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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…