FDA-approved single-dose gene therapy for Hemophilia B reduces bleeding episodes by 71%.

Researchers have developed a promising single-dose gene therapy for Hemophilia B, which has shown a 71% reduction in bleeding episodes in clinical trials. The therapy, approved by the FDA in April 2024, enables the liver to produce clotting factor IX, addressing the genetic disorder's symptoms. This breakthrough could significantly enhance patients' quality of life by eliminating the need for regular prophylactic injections, potentially transforming treatment approaches.

September 25, 2024
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