FARA is excited to announce the Center for Experimental Neurotherapeutics at St. Jude Children's Research Hospital in Memphis, TN as the latest site to join the Collaborative Clinical Research Network in Friedreich's Ataxia (CCRN in FA). The CCRN in FA is an international network of clinical research centers that work together to advance treatments and clinical care for individuals with Friedreich's ataxia. Each of the CCRN in FA sites is a place where FA patients can go to see a team of researchers, physicians, and health care providers dedicated to FA.

The CCRN in FA site at St Jude is led by a specialist in pediatric neurological disorders- Dr. Richard Finkel. Dr. Finkel is an international expert in spinal muscular atrophy and played a vital role in developing the first successful therapy for this disease, and he has been a leader in organizing key clinical trials for neuromuscular diseases. He has nearly 30 years of experience in translational research into the biology and treatment of neurogenetic disorders in addition to SMA, including Duchenne muscular dystrophy and Pompe disease.

The CCRN in FA is funded by FARA. The research centers involved in this network, including St. Jude, run a study called Clinical Outcome Measures in Friedreich’s Ataxia (FACOMS). This is a natural history study with the following objectives:
  • Collect data every year to understand progression of FA and quantify change in progression over time
  • Develop and validate outcome measures (timed walk tests, vision tests) to use in future clinical trials
  • Design and efficiently implement effective clinical trials
  • Collect biological samples such as cheek swabs and blood to identify and study biomarkers involved with the disease process and quantify the change in these markers over time
If you would like to participate in this study at St. Jude, please contact the study coordinator Colin Quillivan.