Gene Therapy for Hemophilia: A Breakthrough Journey

April 18, 2025

Gene therapy for hemophilia has emerged as a groundbreaking advancement in the treatment of this bleeding disorder, particularly for patients suffering from hemophilia B.This innovative approach, exemplified by the FDA-approved Hemgenix gene therapy, aims to correct the underlying genetic defect that leads to a deficiency in clotting factor IX, a crucial element in blood coagulation.
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