Friedreich Ataxia — Research Summary
Printed from RareWays (rareways.com.au) on 27 July 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
RTA‑408 induces p38‑dependent apoptosis and suppresses cell viability in hepatocellular carcinoma cells.
Chen Wei-Chieh et al. — Hepatic oncology (1 December 2026)
https://pubmed.ncbi.nlm.nih.gov/42041104/
- 2.
Neurophysiological assessment of disease severity in Friedreich's Ataxia: a study of brainstem auditory and visual evoked potentials.
Maccora Simona et al. — Clinical neurophysiology : official journal of the International Federation of Clinical Neurophysiology (1 August 2026)
https://pubmed.ncbi.nlm.nih.gov/42143837/
- 3.
Dysregulation of sphingolipid-metabolizing enzymes in Friedreich's ataxia:
Ramchunder Zenouska et al. — iScience (17 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42383006/
- 4.
Interleukin-6: A Potential Link in the Pathophysiology of Restless Legs Syndrome.
Kalkman Hans O et al. — Immunology letters (2 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42392513/
- 5.
Frataxin attenuates endothelial inflammation triggered by engulfment of senescent erythrocytes.
Cheng Xingyi et al. — Free radical biology & medicine (2 July 2026)
https://pubmed.ncbi.nlm.nih.gov/42392282/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Recruiting — Sanford Health
https://clinicaltrials.gov/study/NCT01793168
- 2.
A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
Recruiting — Phase 3 — Biogen
https://clinicaltrials.gov/study/NCT06953583
- 3.
Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Recruiting — Friedreich's Ataxia Research Alliance
https://clinicaltrials.gov/study/NCT06016946
- 4.
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
Recruiting — Phase 1 — Design Therapeutics, Inc.
https://clinicaltrials.gov/study/NCT06874010
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Friedreich Ataxia
Friedreich ataxia is a progressive genetic condition that damages the nervous system and heart, causing loss of coordination, balance problems, and heart disease. It is the most common inherited ataxia. Symptoms usually begin in childhood or adolescence. The first approved treatment, omaveloxolone, reached approval in 2023.
Most Recent Research
INTRODUCTION: Hepatocellular carcinoma (HCC) remains difficult to treat, highlighting the need for new therapeutic strategies. RTA-408 (omaveloxolone), a synthetic oleanane triterpenoid and NRF2 pathway modulator, has reported anticancer activity, but its mechanisms in HCC are not fully understood. MATERIALS AND METHODS: HepG2 and PLC/PRF/5 (PP5) cells were treated with RTA-408 for 24 h. Cell viability, apoptosis, and signaling pathways were evaluated using MTT assay, Annexin V/7-AAD flow cytometry, and western blotting. The role of p38 signaling was examined using the p38 inhibitor SB203580. RESULTS: RTA-408 reduced cell viability in a concentration-dependent manner and increased apoptosis in both cell lines. At 600 nM, apoptosis increased to approximately 18.43% in HepG2 cells and 24.71% in PP5 cells. RTA-408 increased p38 phosphorylation and NRF2 expression and was accompanied by LC3B and p62 accumulation and elevated cleaved caspase-3. Inhibition of p38 partially restored cell viability and reduced apoptosis. CONCLUSION: RTA-408 suppresses HCC cell survival through a p38-dependent stress response associated with NRF2 activation and LC3B/p62 accumulation.
Common Questions
What is Friedreich Ataxia?
Friedreich ataxia is a progressive genetic condition that damages the nervous system and heart, causing loss of coordination, balance problems, and heart disease. It is the most common inherited ataxia. Symptoms usually begin in childhood or adolescence. The first approved treatment, omaveloxolone, reached approval in 2023.
How many clinical trials are available for Friedreich Ataxia?
RareWays currently indexes 122 clinical trials for Friedreich Ataxia, of which 19 are actively recruiting. Trial availability changes as new studies are registered — check the trials tab for current status.
Where does the research data for Friedreich Ataxia come from?
RareWays aggregates research from PubMed, Europe PMC, OpenAlex, and ClinicalTrials.gov. Data is updated regularly by Rocky, RareWays' automated research engine. All articles and trials link directly to their original sources.
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This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.