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.

Read about the prevalence of inherited thrombophilias (IT), potential benefits and limitations of IT testing in healthy children, and recommendations from Children Hospital's experts.

Recent gene therapy trial for hemophilia B showed promising results, with patients’ average factor IX level of 32 percent after treatment. CHOP aims to open a hemophilia A gene therapy trial modeled after the hemophilia B trial.

One of our HTC families created a fundraising project to support hemophilia research through the CHOP Foundation. The response has been so terrific that Zoe, the sister of our patient “Iron Max,” has upped the fundraising goal multiple times.

Genetic testing can help us better estimate risk for inhibitor formation, since inhibitors are more common with certain mutations causing hemophilia.