FA-ICD: Regulatory-Grade Evidence for a Rare Disease References

Case Study: Friedreich’s Ataxia Integrated Clinical Database: Regulatory-Grade Evidence for a Rare Disease

1. Lynch DR, Goldsberry A, Rummey C, et al. Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data. Ann Clin Transl Neurol. 2024;11(1):4-16. doi:10.1002/acn3.51897
2. Lynch DR, Chin MP, Delatycki MB, et al. Safety and efficacy of omaveloxolone in Friedreich ataxia (MOXIe Study). Ann Neurol. 2021;89(2):212-225. doi:10.1002/ana.25934
3. Lynch DR, Chin MP, Boesch S, et al. Efficacy of omaveloxolone in Friedreich’s ataxia: delayed-start analysis of the MOXIe extension. Mov Disord. 2023;38(2):313-320. doi:10.1002/mds.29286
4. US Food and Drug Administration. FDA approves first treatment for Friedreich’s ataxia. February 28, 2023.
5. Critical Path Institute, Friedreich’s Ataxia Research Alliance. C-Path and FARA launch Friedreich’s Ataxia Integrated Clinical Database to advance the development of treatments for FA. February 27, 2019.
6. Critical Path Institute. C-Path partners with FARA to fortify RDCA-DAP and further accelerate drug development with new Friedreich’s ataxia data. 2024.
7. Rummey C, Corben LA, Delatycki MB, et al. Evaluating mFARS in pediatric Friedreich’s ataxia: insights from the FACHILD study. Ann Clin Transl Neurol. 2024. doi:10.1002/acn3.52057
8. Patel M, Isaacs CJ, Seyer L, et al. Progression of Friedreich ataxia: quantitative characterization over 5 years. Ann Clin Transl Neurol. 2016;3(9):684-694.
9. Rummey C, Corben LA, Delatycki M, et al. Natural history of Friedreich ataxia: heterogeneity of neurologic progression and consequences for clinical trial design. Neurology. 2022;99(14):e1499-e1510.