Here you will find a summary of recently published and ongoing trials. This summary is not exhaustive, but illustrates that a number of types of drug trials have been completed, or are currently taking place. There are also many more trials in the pipeline, either to confirm the results of pilot studies listed or exploring new potential treatments (see www.clinical trials.gov for clinical trials information).
This table was updated in June 2026.
| Treatment | Ataxia type | Investigator or company | Clinical stage and clinical research references |
| Omaveloxolone | Friedreich’s ataxia | Biogen | Phase 3 published [1, 2, 3] |
| Etravirine (drug repurposing) | Friedreich’s ataxia | Scientific Institute IRCCS, Italy | Phase 2 published [4] |
| Vatiquinone | Friedreich’s ataxia | PTC Therapeutics | FDA did not approve in 2026. PTC plans to conduct another trial. |
| Nomlabofusp | Friedreich’s ataxia | Larimar | Open label study ongoing in US |
| LX2006 (gene therapy) | Friedreich’s ataxia | Lexeo Therapeutics | Phase 1/2 ongoing in US |
| SGT-212 (gene therapy) | Friedreich’s ataxia | Solid Biosciences | Phase 1b ongoing in US |
| DT219P2 | Friedreich’s ataxia | Design Therapeutics | Phase 1/2 ongoing in Australia |
| Transcranial stimulation (multiple types) | Mixed ataxias | Multiple | Multiple studies with mixed results [5, 6, 7, 8, 9, 10, 11, 12] |
| 4-aminopyridine (4-AP) | SCA27B | Solaxa and an investigator-led study at Assistance Publique - Hôpitaux de Paris, France | Phase 3 ongoing, other clinical results and observations published [13, 14, 15] |
| ARO-ATXN2 (gene silencing) | SCA2 | Arrowhead Therapeutics | Phase 1 ongoing worldwide, not yet including sites in UK |
| VO659 (antisense oligonucleotide) | SCA1 and SCA3 | VICO Therapeutics | Phase 1/2a ongoing including site in UK |
| Troriluzole | SCAs | Biohaven | Phase 3 trial complete, discussions with FDA ongoing. |
References
| [1] | D. R. Lynch, M. P. Chin, S. Boesch, M. B. Delatycki, P. Giunti, A. Goldsberry, J. C. Hoyle, C. Mariotti, K. D. Mathews, W. Nachbauer, M. O'Grady, S. Perlman, S. H. Subramony, G. Wilmot, T. Zesiewicz and C. J. Meyer, “Efficacy of Omaveloxolone in Friedreich's Ataxia: Delayed-Start Analysis of the MOXIe Extension,” Movement Disorders, vol. 38, no. 2, pp. 313-320, 2023. |
| [2] | D. R. Lynch, M. P. Chin, M. B. Delatycki, S. H. Subramony, M. Corti, J. C. Hoyle, S. Boesch, W. Nachbauer, C. Mariotti, K. D. Mathews, P. Giunti, G. Wilmot, T. Zesiewicz, S. Perlman, A. Goldsberry, M. O'Grady and C. J. Meyer, “Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study),” Annals of Neurology, vol. 89, no. 2, pp. 212-225, 2021. |
| [3] | D. R. Lynch, A. Goldsberry, C. Rummey, J. Farmer, S. Boesch, M. B. Delatycki, P. Giunti, J. C. Hoyle, C. Mariotti, K. D. Mathews, W. Nachbauer, S. Perlman, S. H. Subramony, G. Wilmot, T. Zesiewicz, L. Weissfeld and C. Meyer, “Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data,” Annals of clinical and translational neurology, vol. 11, no. 1, pp. 4-16, 2024. |
| [4] | G. Paparella, C. Straga, N. Pesenti, V. D. Molin, G. A. Martorel, V. Merotto, C. Genova, A. Piazza, G. Piccoli, E. Panzeri, A. Rufini, R. Testi and A. Martinuzzi, “A Pilot Phase 2 Randomized Trial to Evaluate the Safety and Potential Efficacy of Etravirine in Friedreich Ataxia Patients,” Children (Basel), vol. 11, no. 8, p. 958, 2024. |
| [5] | Y. Shi, G. Zou, Z. Chen, L. Wan, L. Peng, H. Peng, L. Shen, K. Xia, R. Qiu, B. Tang and H. Jiang, “Efficacy of cerebellar transcranial magnetic stimulation in spinocerebellar ataxia type 3: a randomized, single-blinded, controlled trial,” Journal of Neurology, vol. 270, no. 11, pp. 5372-5379, 2023. |
| [6] | R. P. P. W. M. Maas, S. Teerenstra, I. Toni, T. Klockgether, D. J. L. G. Schutter and B. P. C. van de Warrenburg, “Cerebellar Transcranial Direct Current Stimulation in Spinocerebellar Ataxia Type 3: a Randomized, Double-Blind, Sham-Controlled Trial,” Neurotherapeutics, vol. 19, no. 4, pp. 1259-1272, 2022. |
| [7] | G. Naeije, A. Rovai, V. Destrebecq, N. Trotta and X. De Tiege, “Anodal Cerebellar Transcranial Direct Current Stimulation Reduces Motor and Cognitive Symptoms in Friedreich's Ataxia: A Randomized, Sham-Controlled Trial,” Movement Disorders, vol. 38, no. 8, pp. 1443-1450, 2023. |
| [8] | I. Libri, V. Cantoni, A. Benussi, J. Rivolta, C. Ferrari, R. Fancellu, M. Synofzik, A. Alberici, A. Padovani and B. Borroni, “Comparing Cerebellar tDCS and Cerebellar tACS in Neurodegenerative Ataxias Using Wearable Sensors: A Randomized, Double-Blind, Sham-Controlled, Triple-Crossover Trial,” Cerebellum, vol. 23, no. 2, pp. 570-578, 2024. |
| [9] | R. Brito, J. V. Fabricio, A. Araujo, G. Barreto, A. Baltar and K. Monte-Silva, “Single-Session Cerebellar Transcranial Direct Current Stimulation Improves Postural Stability and Reduces Ataxia Symptoms in Spinocerebellar Ataxia,” Cerebellum, vol. 23, no. 5, pp. 1993-2002, 2024. |
| [10] | X. Chen, X. Liu, W. Lin, L. Zhang, X. Cheng, Z. Huang, W. Zhang, H. Zeng, Y. Lian, Y. Zhang, M. Li, N. Chen, S. Huang, Z. Wang, X. Wang, Z. Liu, R. Yuan, X. Chen, Z. Ye, B. Cheng, Y. Zhang, Q. Chen, D. Wang, J. Ni, N. Wang, Y. Fu and S. Gan, “Transcranial alternating current stimulation for treating spinocerebellar ataxia type 3: A randomized controlled trial,” Cell reports. Medicine, vol. 6, no. 6, p. 102162, 2025. |
| [11] | M. Grobe-Einsler, F. Bork, A. Faikus, R. Hurlemann and O. Kaut, “Effects of cerebellar repetitive transcranial magnetic stimulation plus physiotherapy in spinocerebellar ataxias - A randomized clinical trial,” CNS neuroscience and therapeutics, vol. 30, no. 6, p. e14797, 2024. |
| [12] | A. Sanna, M. Pau, M. Porta, G. Pilia, V. Secci, E. Cartella, A. Demattia, A. Paribello, G. Cossu, A. Milia and P. Tacconi, “Efficacy of Cerebellar Transcranial Direct Current Stimulation in Degenerative Ataxia. A Sham-Controlled Clinical and Quantitative Analysis,” Cerebellum, vol. 25, no. 1, p. 11, 2026. |
| [13] | J. Seemann, A. Traschutz, W. Ilg and M. Synofzik, “4-Aminopyridine improves real-life gait performance in SCA27B on a single-subject level: a prospective n-of-1 treatment experience,” Journal of Neurology, vol. 270, no. 11, pp. 5629-5634, 2023. |
| [14] | D. Pellerin, F. Heindl, C. Wilke, M. C. Danzi, A. Traschutz, C. Ashton, M.-J. Dicaire, A. Cuillerier, G. Del Gobbo, K. M. Boycott, J. Claassen, D. Rujescu, A. M. Hartmann, S. Zuchner, B. Brais, M. Strupp and M. Synofzik, “GAA-FGF14 disease: defining its frequency, molecular basis, and 4-aminopyridine response in a large downbeat nystagmus cohort,” EBioMedicine, vol. 102, no. 105076, 2024. |
| [15] | C. Wilke, D. Pellerin, D. Mengel, A. Traschutz, M. C. Danzi, M.-J. Dicaire, M. Neumann, H. Lerche, B. Bender, H. Houlden, S. Zuchner, L. Schols, B. Brais and M. Synofzik, “GAA-FGF14 ataxia (SCA27B): phenotypic profile, natural history progression and 4-aminopyridine treatment response,” Brain, vol. 146, no. 10, pp. 4144-4157, 2023. |
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