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For Phil, his hemophilia diagnosis in 1970 came as both a relief and a shock. He was grateful to finally understand what he was experiencing, but at the same time, prognosis was grim, and the treatment landscape was limited. The introduction of extended half-life therapy 10 years ago was an important milestone for people like Phil. Then and now, we’re committed to supporting the hemophilia community by listening to their needs, developing new treatment options, and helping to address the ongoing
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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia. Read more: http://www.nejm.org/doi/full/10.1056/NEJMoa1708538#t=abstract | Blood and Hematology
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