Hemophilia and Bleeding Disorders Program News

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CHOP-led Study Shows Novel Gene Therapy for Hemophilia A Leads to Sustained Expression of Clotting Factor and Reduced Bleeding Events

Nov 17, 2021

Results from multicenter study are first to show improved production of coagulation factor VIII over prolonged period, leading to reduction or complete elimination of bleeding events.

CHOP Gene Therapy Experts Profiled in Philadelphia Magazine

May 12, 2021

CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.

CHOP-led Research Study Identifies Key Target in Treatment-Resistant Hemophilia A

Apr 15, 2021

CHOP researchers have identified BAFF as a key target that may be responsible for treatment failure in about 30% of patients with hemophilia A.

Researchers Uncover A Previously Underappreciated Mechanism that Turns Off Clotting Factor in Hemophilia A

Jan 13, 2021

CHOP researchers identified an in vivo role for activated protein C (APC), which could potentially be exploited for future hemophilia A treatments.

Gene-based Clotting Factor Prevents Bleeding in Animals with Hemophilia

Apr 15, 2019

A laboratory study may set the stage for a new preventive treatment for hemophilia patients who now develop antibodies to the standard treatment and then require a bypass therapy.

CHOP Investigator and Experimental One-dose Gene Therapy for Hemophilia B Featured in The New York Times

Aug 13, 2018

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

Dec 7, 2017

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.

3 CHOP Scientists Win Bayer Grants for Hemophilia Research

Jul 27, 2017

Three CHOP researchers recently won grants for their work on hemophilia from the Bayer Hemophilia Awards Program.

The Importance of Genetic Testing for Hemophilia

Jul 1, 2013

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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