Q: Friedreich's Ataxia Research Alliance (FARA) very early on launched a natural history study of the disease. Can you describe the scope and purpose of it?
FARA and clinical researchers established the Collaborative Clinical Research Network (CCRN) in Friedreich's ataxia in about 2004, with the initial goal of developing clinical endpoints for Friedreich’s ataxia (FA). At that point, no industry-sponsored clinical trials had been carried out in FA, and FARA recognized that the community would need established clinical endpoints and clinical research infrastructure in order to be able to advance therapeutic development. These early studies to evaluate clinical outcome measures and disease-specific rating scales became the basis for an ongoing natural history study that has collected data on over 850 patients. Today the goals of the CCRN include:
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