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.
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.
CHOP's Transition to Adulthood Program website has a Resources section that includes age-specific tip sheets for parents or guardians and for children and youth, videos and information about in-person workshops that are hosted for families and teens.
As part of our ongoing goal to provide the best care possible and to collect required data, all patients between the ages of 12 and 22 will be asked to complete a patient experience survey when you come to clinic for your annual comprehensive care visit.