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Hemophilia B - Single Gene Therapy Treatment Offers Significant Improvement

Patients with hemophilia B experienced considerable improvements and fewer injections with clotting factor to reduce bleeding after receiving just one treatment with gene therapy, researchers from St. Jude Children's Research Hospital, Memphis, USA, and University College London (UCL), England, reported in NEJM (New England Journal of Medicine).

The small Phase I study, involving just six patients, is still proof that gene therapy is a promising treatment for the disabling and potentially dangerous consequences of painful bleeding episodes experienced by patients with hemophilia B - an inherited blood disorder.

The authors say their study will be presented in San Diego, USA, on 11th December, 2011, at the 53rd annual meeting of the American Society of Hematology.

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