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Leslie Raffini, MD, and Regina Butler, RN, were quoted in the Philadelphia Inquirer on a story featuring a CHOP patient family living with hemophilia, an inherited bleeding disorder that prevents blood from clotting properly.
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Dr. George was recognized for the first gene therapy clinical trial to report a cure of hemophilia B patients.
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CHOP breakthrough helps scientists edge closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots.
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A team of gene therapy researchers has reported on a highly active gene variant that achieved sustained factor levels in 10 adults, with no adverse effects.
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Genetic testing can help us better estimate risk for inhibitor formation, since inhibitors are more common with certain mutations causing hemophilia.
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Children and adolescents with bleeding and clotting disorders (who may be on anticoagulation therapy), picking a sport requires special consideration to minimize risks of bleeding.
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In December 2012, CHOP opened a gene therapy clinical trial for patients older than 18 years of age, with factor IX levels equal to or less than 2 percent.