CHOP Hematologist Dr. Lindsey George Receives 2019 Clinical Achievement Research Award
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Dr. George was recognized for the first gene therapy clinical trial to report a cure of hemophilia B patients.
Hemophilia News and Updates
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CHOP Investigator and Experimental One-dose Gene Therapy for Hemophilia B Featured in The New York Times
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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.
One-dose Gene Therapy Produces Clotting Factor, Safely Stops Bleeding in Hemophilia B Patients
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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.
Washington Post Reports on Hemophilia B Gene Therapy Advance
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A clinical trial of a gene therapy for hemophilia B is allowing patients to avoid painful, disabling internal bleeds.
After One Dose of Gene Therapy Hemophilia B Patients Maintain Near-normal Levels of Clotting Factor
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In a clinical trial led by a CHOP researcher, adult patients with hemophilia B were able to pursue daily activities without disabling bleeds.
CHOP Scientist Honored for Gene Therapy Research
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CHOP’s Katherine High, MD, was honored for her trailblazing research in the bleeding disorder hemophilia with a national award from the American Society of Hematology.
Decoy Stops Immune System From Defeating Hemophilia Therapy
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Gene therapy researchers from The Children's Hospital of Philadelphia created a decoy to fool the immune system, preventing it from mistakenly defeating the benefits delivered by a corrective gene.
The Importance of Genetic Testing for Hemophilia
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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.
Safety, Sports and Activity Limits
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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.
Gene Therapy for Patients with Factor IX Deficiency
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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.