Scientific Programs

Novel Therapies for Hemophilia – Gene Therapy Replacement

During Tuesday’s State-of-the-Art Session “Novel Therapies for Hemophilia”, President and Chief Scientific Officer of Spark Therapeutics, Katherine High, M.D., Ph.D. summarized findings from their phase I/II trial of the long-term safety and efficacy of factor IX (FIX) gene therapy in patients with hemophilia B

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