At Children’s Hospital of Philadelphia’s Friedreich’s Ataxia Program, children and young adults with Friedreich's ataxia have access to top-notch clinical care and cutting-edge research opportunities in one place.
More FA patients and families come here than anywhere else in the world to find the best clinical care available, participate in promising clinical trials, and learn about the exciting FA research going on here and around the world.
As the largest FA clinical program in the world, we are uniquely equipped to care for children with this rare, complex disease.
A Center of Excellence
CHOP’s FA Program is part of the Friedreich’s Ataxia Center of Excellence, a first-of-its-kind program dedicated to make a cure a reality.
Established in March 2014 by Children’s Hospital of Philadelphia, Penn Medicine, and the Friedreich’s Ataxia Research Alliance (FARA), the center is dedicated to fostering FA research, clinical care, drug discovery and development.
This collaboration enables us to provide the unique, specialized care needed by FA patients, and supports our goal of advancing treatment of the disease with more regular collaboration between scientists and physicians.
A commitment to research
CHOP’s FA Program is a key player in the highly collaborative international FA research community. CHOP, FARA and Penn Medicine have long been at the forefront of the study of FA, collaborating on research and clinical trials to pinpoint the metabolic dysfunction at its root while managing the largest natural history database in rare disease today.
Working with pharmaceutical partners, a pipeline of more than 20 drug candidates is ready to be mined for new therapies. Several drug trials are currently underway, many of which have shown some progress at improving symptoms or slowing the progression of the disease.
Between these pioneering research efforts and our large patient population, the Friedreich’s Ataxia Center of Excellence is in a unique position to advance our knowledge of this disease and inform future research.
With 300 patients, the clinical program at CHOP is the largest in the world and serves patients throughout their lives through the close partnership with Penn Medicine. The program’s youngest patient is 4; its oldest is 83.
At CHOP’s FA Program, you will find a “home” for you or your child’s care. From neurology to the many different specialists often needed in FA — including cardiology, orthopedics and endocrinology for diabetes care — everything is coordinated by one central program.