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FRDA is a multisystem disorder involving the central and peripheral nervous systems, the musculoskeletal system, the heart, and the endocrine pancreas. In recent years, Omaveloxolone, a potent activator of nuclear factor erythroid 2-related factor 2 signaling, showed a significant neurological improvement compared to placebo, with a good safety profile. With this study, we report an early real-life experience on a cohort of FRDA patients treated with omaveloxolone.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Materials and methods<\/jats:title>\n                    <jats:p>Patients were assessed with an anamnestic profile, general and neurological examination, clinical scales (mFARS, SARA, and FA-ADL) and blood tests, at baseline, at 12\u00a0weeks and after 24\u00a0weeks of treatment. Inclusion criteria were genetical diagnosis of FRDA, age\u2009\u2265\u200918\u00a0years and mFARS\u2009&lt;\u200980. Exclusion criteria included severe hepatic and renal impairment, and severe heart failure. Each patient received oral omaveloxolone at a dose of 150\u00a0mg\/day.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Twenty patients (65% females) affected by FRDA aged 40.6\u2009\u00b1\u200912.6\u00a0years and a duration of disease of 24.9\u2009\u00b1\u20099.5\u00a0years were treated with omaveloxolone and followed up for 25.2\u2009\u00b1\u20098.0\u00a0weeks. The drug was safe with no significant adverse events during the first 24\u00a0weeks and without discontinuations. Indeed, asymptomatic, and transient liver transaminase elevation occurred in 50% of patients. Cardiac function was stable, as well as NT-proBNP and lipids. Clinical scales did not show any significant difference during follow-up, but a significant reduction in IL-6 was reported.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions and discussion<\/jats:title>\n                    <jats:p>Omaveloxolone\u00a0seems to be safe and well-tolerated in adult FRDA patients in the real-life setting. No significant worsening of symptoms was observed with no signs of progression, as well as the improvement of inflammatory biomarkers after 24\u00a0weeks of treatment, but no predictive factors for the disease response have been identified. However, the short duration, and the small sample size limit the generalizability of the results. Further studies with longer observation are needed to clearly define the efficacy of omaveloxolone\u00a0in FRDA.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1007\/s00415-025-13487-1","type":"journal-article","created":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T18:23:07Z","timestamp":1762021387000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Early experience on omaveloxolone in adult patients with Friedreich\u2019s ataxia: a real-world observational study"],"prefix":"10.1007","volume":"272","author":[{"given":"Salvatore Maria","family":"Lima","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marta","family":"Caltagirone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Messina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Umberto","family":"Quartetti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicasio","family":"Rini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Flora","family":"D\u2019Amico","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Filippo","family":"Brighina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9805-1655","authenticated-orcid":false,"given":"Vincenzo","family":"Di Stefano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,11,1]]},"reference":[{"issue":"1","key":"13487_CR1","doi-asserted-by":"publisher","first-page":"19","DOI":"10.1093\/bmb\/ldx034","volume":"124","author":"A Cook","year":"2017","unstructured":"Cook A, Giunti P (2017) Friedreich\u2019s ataxia: clinical features, pathogenesis and management. 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