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The specific aims must target one or more of the following:
1. Identification of biomarkers for FA that will elucidate disease variability, severity, and prognosis; facilitate drug screening, and/or optimize selection of patients and clinical endpoints for clinical trials;
2. Development of tools and technologies that can (a) be directly used for therapy development; (b) overcome existing obstacles to treatment and (c) be directly applied to, or adapted for, delivery of potential therapeutics;
3. Pre-clinical development and testing of potential therapeutics, biologics, and devices in cells and animals;
4. Clinical studies of patient outcome measures, potential interventions, or devices.