772
Views
0
CrossRef citations to date
0
Altmetric
Editorial

Experimental drugs for Friedrich’s ataxia: progress and setbacks in clinical trials

ORCID Icon & ORCID Icon
Pages 967-969 | Received 21 Sep 2023, Accepted 25 Oct 2023, Published online: 30 Oct 2023

References

  • Indelicato E, Nachbauer W, Eigentler A, et al. Onset features and time to diagnosis in Friedreich’s Ataxia. Orphanet J Rare Dis. 2020;15(1). doi: 10.1186/s13023-020-01475-9
  • Campuzano V, Montermini L, Moltò MD, et al. Friedreich’s ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion. Science. 1996;80-) 271(5254):1423–1427. doi: 10.1126/science.271.5254.1423
  • Reetz K, Dogan I, Hilgers RD, et al. Progression characteristics of the European Friedreich’s Ataxia consortium for translational studies (EFACTS): a 2 year cohort study. Lancet Neurol. 2016;15(13):1346–1354. doi: 10.1016/S1474-4422(16)30287-3
  • 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. doi: 10.1212/WNL.0000000000200913
  • Tsou AY, Paulsen EK, Lagedrost SJ, et al. Mortality in Friedreich Ataxia. J Neurol Sci. 2011;307(1–2):46–49. doi: 10.1016/j.jns.2011.05.023
  • Indelicato E, Bösch S. Emerging therapeutics for the treatment of Friedreich’s ataxia. Expert Opin Orphan Drugs. 2018;6(1):57–67. doi: 10.1080/21678707.2018.1409109
  • Rodden LN, Lynch DR. Designing phase II clinical trials in Friedreich ataxia. Expert Opin Emerg Drugs. 2021;26(4):415–423. doi: 10.1080/14728214.2021.1998452
  • Lee A. Omaveloxolone: First Approval. Drugs. 2023;83(8):725–729. doi: 10.1007/s40265-023-01874-9
  • Paupe V, Dassa EP, Goncalves S, et al. Impaired nuclear Nrf2 translocation undermines the oxidative stress response in Friedreich Ataxia. PLoS One. 2009;4(1):e4253. doi: 10.1371/JOURNAL.PONE.0004253
  • 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
  • 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
  • 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. doi: 10.1002/ACN3.332
  • Weber SC. The evolution and use of patient-reported outcomes in regulatory decision making. Regul Focus Q. 2023;3:4–9.
  • Pandolfo M, Reetz K, Darling A, et al. Efficacy and safety of Leriglitazone in patients with Friedreich Ataxia: a phase 2 double-blind, randomized controlled trial (FRAMES). Neurol Genet. 2022;8(6):e200034. doi: 10.1212/NXG.0000000000200034
  • Sivakumar A, Cherqui S. Advantages and limitations of gene therapy and gene editing for Friedreich’s Ataxia. Front Genome Ed. 2022;4:903139. doi: 10.3389/fgeed.2022.903139
  • Babcock M, De Silva D, Oaks R, et al. Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin. Science. 1997;80-) 276(5319):1709–1712. doi: 10.1126/science.276.5319.1709
  • Pandolfo M, Arpa J, Delatycki MB, et al. Deferiprone in Friedreich ataxia: a 6-month randomized controlled trial. Ann Neurol. 2014;76(4):509–521. doi: 10.1002/ana.24248
  • 2023Hyperkinetic Movement DisordersMov DisordVol. 3810.1002/MDS.29542
  • Indelicato E, Kirchmair A, Amprosi M, et al. Skeletal muscle transcriptomics dissects the pathogenesis of Friedreich’s ataxia. Hum Mol Genet. 2023;32(13):2241–2250. doi: 10.1093/HMG/DDAD051
  • C-Path and FARA Launch Friedreich’s Ataxia integrated clinical database to advance the development of treatments for FA. [cited 2019 Feb 27]. https://c-path.org/c-path-and-fara-launch-friedreichs-ataxia-integrated-clinical-database-to-advance-the-development-of-treatments-for-fa/

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.