CHOP Hematologist Dr. Lindsey George Receives 2019 Clinical Achievement Research Award
Published on in CHOP News
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
Published on in CHOP News
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
Published on in CHOP News
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
Published on in CHOP News
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
Published on in CHOP News, International Update
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
Published on in CHOP News
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
Published on in CHOP News
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
Published on in Beyond Blood
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
Published on in Beyond Blood
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
Published on in Beyond Blood
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.