Gene Therapy for Hemophilia: A New Hope for Patients

April 18, 2025

Gene therapy for hemophilia represents a groundbreaking advancement in treating this genetic disorder, particularly hemophilia B, where patients suffer from a deficiency of clotting factor IX.In February 2025, Terence Blue became the first individual in New England to receive Hemgenix, a pioneering gene therapy approved by the FDA in 2022, specifically designed to address the root cause of hemophilia.
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