Learning your child has a progressive condition like Friedreich's ataxia (FA) can be very upsetting. We are here for you.

More patients and families come to the Friedreich’s Ataxia Program at Children's Hospital of Philadelphia (CHOP) than anywhere else in the world. They come because we offer the best clinical care available. They come to participate in promising clinical trials. They come to learn about the exciting advances on the horizon.

As the largest Friedreich's ataxia (FA) clinical program in the world, we are uniquely able to care for children with this rare, complex disease.

A commitment to care and research

We know how upsetting it is to learn that, currently, there is no cure for Friedreich's ataxia. At Children's Hospital of Philadelphia Friedreich’s Ataxia Center of Excellence, we focus our energy on doing all that we can to support you and your child, manage the symptoms of Friedreich's ataxia, and improve your child’s quality of life.

In addition to offering prescription medications and over-the-counter supplements that can help manage some of the symptoms of the condition, physical therapy and regular exercise can help you or your child maximize muscle functionality and prolong walking.

Our collaboration with Friedreich’s Ataxia Center of Excellence not only helps advance the treatment of the disease but also allows us to provide the unique, specialized care our patients need. We constantly adjust our approach to care based on the latest research findings and treatment approaches.

The international FA research community is very collaborative. CHOP’s Friedreich's Ataxia Program is a leader in this community. We are part of the Friedreich’s Ataxia Center of Excellence, which was established in March 2014 by CHOP, Penn Medicine and the Friedreich’s Ataxia Research Alliance (FARA). The Center of Excellence is dedicated to finding a cure for FA.

Together, we manage the largest natural history database in rare diseases today. A natural history database is a collection of detailed clinical information about a specific disease or condition, gathered over time. It helps researchers and healthcare professionals understand how the disease progresses and affects patients. We are working with pharmaceutical partners to use this information to develop new therapies. We have several promising drug trials currently underway.

Between these pioneering research efforts and our large patient population, we are in a unique position to advance our knowledge of this disease and inform future research.

Lifelong care

With 300 patients, the clinical program at CHOP is the largest in the world. We serve patients throughout their lives through our close partnership with Penn Medicine. Our youngest patient is 4, and our oldest patient is 83.

At CHOP, you will find a comforting environment 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.