Every year, the Friedreich’s Ataxia Program at The Children's Hospital of Philadelphia holds a one-day symposium to provide patients and families with up-to-date clinical information, therapeutic approaches and current research being conducted in the field of Friedreich’s ataxia (FA). The Eighth Annual Friedreich’s Ataxia Symposium was held on Oct. 12, 2015.
Topics included: clinical management and neurologic dysfunction; FA drug discovery and treatment pipeline; updates on a variety of research projects including the FARA patient registry, RT001 trial, MOXIe trial (RTA 408), STEADFAST trial (ACTIMMUNE), gene therapy and more; cardiac function and exercise; muscle spectroscopy; and college resources and accessibility.