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Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children’s Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K. The findings of the six-person study mark the first proof that gene therapy can reduce disabling, painful bleeding episodes in patients with the inherited blood disorder. Results of the Phase I study appear in the December 10 online edition of the New England Journal of Medicine. The research is also scheduled to be presented December 11 at the 53rd annual meeting of the American Society of Hematology in San Diego. To view Multimedia News Release, go to http://www.multivu.com/mnr/46766-st-jude-children-s-research-hospital-gene-therapy-bleeding-disorder

Today on Rare Disease Day, My Life, Our Future, a national program founded by leaders in the bleeding disorder community, including Bloodworks Northwest, the American Thrombosis and Hemostasis Network, the National Hemophilia Foundation and Bioverativ Inc., opened the largest research repository of its kind in the world to scientists. The My Life, Our Future Research Repository is a collection of genetic data and blood samples that are linked to phenotypic data from more than 5,000 people in the U.S. with hemophilia, a rare disorder that impairs the ability of one’s blood to clot. The My Life, Our Future Research Repository will allow researchers to advance the scientific understanding of the disorder, including genetic differences that affect bleeding severity and reaction to certain treatments. To view the multimedia release go to: https://www.multivu.com/players/English/8046151-national-hemophilia-foundation-my-life-our-future/