Long-Term follow-up of the NIH Friedreich’s Ataxia Idebenone Study Cohort
Principal Investigator: Kimberly Lin, MD, Children’s Hospital of Philadelphia
This study is to retrospectively assess disease progression in Friedreich’s ataxia (FRDA) patients. We will contact individuals with FRDA who previously participated in 05-N-0245 at the NIH. If these former subjects decide to enroll in the study and provide their informed consent, we will collect from them preexisting documentation and records of information on their cardiac status.
Progression in measures will be tabulated using baseline visit data from 05-N-0245. The study will compare different measures of disease to identify areas of change. The study will also attempt to identify certain factors that predict rate of progression and disease outcomes. Information will be collected using obtained medical records (including most recent ECHO, EST, CMR, and biomarker evaluations) and data collected in the context of recent CCRNinFA study visits and/or clinical encounters.
For more information about this study please contact Felice Wilson at 267-426-2995.