Hemostasis and Thrombosis Center News

Meet physician-scientist Stella T. Chou, MD, the new Chief of the Division of Hematology. Read about the promise of emerging therapies, what makes CHOP unique and more.

Researchers at Children’s Hospital of Philadelphia (CHOP) sought to better understand how the body regulates activated FVIII function, especially in interactions with other clotting factors, to inform associated pathology and therapeutic drug design.

Researchers at Children’s Hospital of Philadelphia (CHOP) announced the results of the longest follow-up study to date for individuals receiving the gene therapy fidanacogene elaparvovec to treat hemophilia B, a rare, X-linked disorder resulting from a deficiency in coagulation factor IX (FIX) activity.

In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.

Family Newsletter from the Comprehensive Hemostasis and Thrombosis Center at Children’s Hospital of Philadelphia.

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.

A look back at the last five decades of treatment options to what lies ahead with additional potentially curative gene therapies.

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

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