{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,27]],"date-time":"2026-10-27T22:53:47Z","timestamp":1793141627512,"version":"4.5.0"},"reference-count":100,"publisher":"Informa UK Limited","issue":"1","content-domain":{"domain":["www.tandfonline.com"],"crossmark-restriction":true},"short-container-title":["Expert Opinion on Investigational Drugs"],"published-print":{"date-parts":[[2023,1,2]]},"DOI":"10.1080\/13543784.2023.2173063","type":"journal-article","created":{"date-parts":[[2023,1,28]],"date-time":"2023-01-28T13:27:11Z","timestamp":1674912431000},"page":"5-16","update-policy":"https:\/\/doi.org\/10.1080\/tandf_crossmark_01","source":"Crossref","is-referenced-by-count":47,"title":["Omaveloxolone: an activator of Nrf2 for the treatment of Friedreich ataxia"],"prefix":"10.1080","volume":"32","author":[{"given":"Victoria","family":"Profeta","sequence":"first","affiliation":[{"name":"Departments of Pediatrics and Neurology, Children\u2019s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kellie","family":"McIntyre","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Neurology, Children\u2019s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"McKenzie","family":"Wells","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Neurology, Children\u2019s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Courtney","family":"Park","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Neurology, Children\u2019s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David R","family":"Lynch","sequence":"additional","affiliation":[{"name":"Departments of Pediatrics and Neurology, Children\u2019s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"301","published-online":{"date-parts":[[2023,2,8]]},"reference":[{"key":"cit0001","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2011.01.010"},{"key":"cit0002","doi-asserted-by":"publisher","DOI":"10.1016\/B978-0-444-51892-7.00017-6"},{"key":"cit0003","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-009-1002-3"},{"key":"cit0004","doi-asserted-by":"publisher","DOI":"10.2147\/JMDH.S292945"},{"key":"cit0005","doi-asserted-by":"publisher","DOI":"10.1002\/1531-8249(199902)45:2<200::AID-ANA10>3.0.CO;2-U"},{"key":"cit0006","doi-asserted-by":"publisher","DOI":"10.1002\/mds.1210"},{"issue":"5","key":"cit0007","first-page":"1251","volume":"60","author":"Bidichandani SI","year":"1997","journal-title":"Am J Hum Genet"},{"key":"cit0008","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2020.264"},{"key":"cit0009","doi-asserted-by":"publisher","DOI":"10.3389\/fphar.2014.00130"},{"key":"cit0010","doi-asserted-by":"publisher","DOI":"10.1038\/ng1097-215"},{"key":"cit0011","doi-asserted-by":"publisher","DOI":"10.1016\/S0969-2126(00)00158-1"},{"key":"cit0012","doi-asserted-by":"publisher","DOI":"10.1002\/1531-8249(199905)45:5<673::AID-ANA20>3.0.CO;2-Q"},{"key":"cit0013","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199610173351601"},{"key":"cit0014","doi-asserted-by":"publisher","DOI":"10.1086\/513887"},{"key":"cit0015","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.55.11.1719"},{"key":"cit0016","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.332"},{"key":"cit0017","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(14)70321-7"},{"key":"cit0018","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2011.05.023"},{"key":"cit0019","doi-asserted-by":"publisher","DOI":"10.1586\/erc.12.57"},{"key":"cit0020","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0116396"},{"key":"cit0021","doi-asserted-by":"publisher","DOI":"10.1093\/ejechocard\/jer267"},{"key":"cit0022","doi-asserted-by":"publisher","DOI":"10.1177\/0883073812448535"},{"key":"cit0023","doi-asserted-by":"publisher","DOI":"10.1016\/j.jns.2017.01.027"},{"key":"cit0024","doi-asserted-by":"publisher","DOI":"10.1016\/j.healun.2003.09.015"},{"key":"cit0025","doi-asserted-by":"publisher","DOI":"10.1016\/j.healun.2016.06.021"},{"key":"cit0026","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2021.01.004"},{"key":"cit0027","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51352"},{"key":"cit0028","doi-asserted-by":"publisher","DOI":"10.1302\/0301-620X.94B5.28391"},{"key":"cit0029","doi-asserted-by":"publisher","DOI":"10.1016\/j.diabres.2022.109828"},{"key":"cit0030","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.686"},{"key":"cit0031","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.439"},{"key":"cit0032","doi-asserted-by":"publisher","DOI":"10.1016\/j.diabres.2018.05.008"},{"key":"cit0033","doi-asserted-by":"publisher","DOI":"10.2337\/diaclin.33.2.84"},{"key":"cit0034","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-013-6978-z"},{"key":"cit0035","doi-asserted-by":"publisher","DOI":"10.3390\/jpm11080708"},{"key":"cit0036","doi-asserted-by":"publisher","DOI":"10.3390\/tomography7040076"},{"key":"cit0037","doi-asserted-by":"publisher","DOI":"10.1002\/mds.25058"},{"key":"cit0038","doi-asserted-by":"publisher","DOI":"10.1111\/jnc.12300"},{"key":"cit0039","doi-asserted-by":"publisher","DOI":"10.3389\/fmolb.2022.830650"},{"key":"cit0040","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(07)70220-X"},{"issue":"8","key":"cit0041","first-page":"CD007791","volume":"2016","author":"Kearney M","year":"2016","journal-title":"Cochrane Database Syst Rev"},{"key":"cit0042","doi-asserted-by":"publisher","DOI":"10.1001\/archneurol.2010.168"},{"key":"cit0043","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.51373"},{"key":"cit0044","doi-asserted-by":"publisher","DOI":"10.2217\/nmt-2022-0011"},{"key":"cit0045","doi-asserted-by":"publisher","DOI":"10.1093\/bmb\/ldx034"},{"key":"cit0046","doi-asserted-by":"publisher","DOI":"10.1186\/s42466-019-0038-9"},{"key":"cit0047","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0214250"},{"key":"cit0048","doi-asserted-by":"publisher","DOI":"10.1007\/s00228-022-03393-0"},{"key":"cit0049","doi-asserted-by":"publisher","DOI":"10.1152\/ajpcell.00243.2006"},{"key":"cit0050","unstructured":"PTC Therapeutics. A randomized, parallel-arm, double-blind, placebo-controlled study with open-label extension to assess the efficacy and safety of vatiquinone for the treatment of Friedreich ataxia (MOVE-FA) [Internet]. clinicaltrials.gov; 2022 [cited 2022 Nov 7]. Report No.: NCT04577352. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04577352."},{"key":"cit0051","doi-asserted-by":"publisher","DOI":"10.1111\/bph.12461"},{"key":"cit0052","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddab232"},{"key":"cit0053","doi-asserted-by":"publisher","DOI":"10.1002\/prp2.755"},{"key":"cit0054","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-017-10320-2"},{"key":"cit0055","doi-asserted-by":"publisher","DOI":"10.1016\/j.nbd.2020.105162"},{"key":"cit0056","unstructured":"eMascar\u00f3. Minoryx\u2019s clinical candidate leriglitazone shows clinical benefit in a proof of concept Phase 2 study in Friedreich\u00b4s ataxia [Internet]. 2022 [cited 2022 Nov 8]. Available from: https:\/\/www.minoryx.com\/media\/minoryx%E2%80%99s_clinical_candidate_leriglitazone_shows_clinical_benefit_in_a_proof_of_concept_phase_2_study_in_friedreichs_ataxia\/."},{"key":"cit0057","doi-asserted-by":"publisher","DOI":"10.1212\/NXG.0000000000200034"},{"key":"cit0058","doi-asserted-by":"publisher","DOI":"10.1016\/j.phrs.2020.104680"},{"key":"cit0059","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(14)60382-2"},{"key":"cit0060","doi-asserted-by":"publisher","DOI":"10.1172\/jci.insight.93885"},{"key":"cit0061","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(14)60573-0"},{"key":"cit0062","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddu408"},{"key":"cit0063","doi-asserted-by":"publisher","DOI":"10.1089\/ars.2012.4537"},{"key":"cit0064","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0217776"},{"key":"cit0065","doi-asserted-by":"publisher","DOI":"10.1126\/science.aan6414"},{"key":"cit0066","unstructured":"Design Therapeutics \u2013 Our Approach [Internet]. 2022 [cited 2022 Nov 8]. Available from: https:\/\/www.designtx.com\/our-approach\/."},{"key":"cit0067","unstructured":"Lexeo Therapeutics. A phase 1\/2 study of the safety and efficacy of LX2006 gene therapy in participants with cardiomyopathy associated with friedreich\u2019s ataxia [Internet]. clinicaltrials.gov; 2022 [cited 2022 Nov 7]. Report No.: NCT05445323. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT05445323."},{"key":"cit0068","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddr554"},{"key":"cit0069","unstructured":"International Congress for Ataxia Research [Internet]. 2022 [cited 2022 Nov 8]. Available from: https:\/\/ataxiacongress.org\/program.htm."},{"key":"cit0070","unstructured":"Lynch D Efficacy of omaveloxolone in patients with friedreich\u2019s ataxia: update of the delayed-start study [Internet]. MDA Clinical & Scientific Conference 2023. [cited 2022 Nov 9]. Available from: https:\/\/www.mdaconference.org\/abstract-library\/efficacy-of-omaveloxolone-in-patients-with-friedreichs-ataxia-update-of-the-delayed-start-study\/."},{"key":"cit0071","unstructured":"Larimar Therapeutics, Inc. A phase 1 single ascending dose study to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of Subcutaneous CTI-1601 Versus Placebo in Subjects With Friedreich\u2019s Ataxia [Internet]. clinicaltrials.gov; 2020 [cited 2022 Nov 7]. Report No.: NCT04176991. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04176991."},{"key":"cit0072","doi-asserted-by":"publisher","DOI":"10.1038\/nm.3510"},{"key":"cit0073","doi-asserted-by":"publisher","DOI":"10.1038\/mtm.2014.44"},{"key":"cit0074","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2018.05.006"},{"key":"cit0075","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2019.363"},{"key":"cit0076","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddy427"},{"key":"cit0077","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2020.08.018"},{"key":"cit0078","doi-asserted-by":"publisher","DOI":"10.3389\/fgeed.2022.903139"},{"key":"cit0079","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-020-17954-3"},{"key":"cit0080","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2020.04.018"},{"key":"cit0081","doi-asserted-by":"publisher","DOI":"10.1016\/j.scr.2019.101529"},{"key":"cit0082","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2015.41"},{"key":"cit0083","doi-asserted-by":"publisher","DOI":"10.1016\/j.phrs.2015.05.015"},{"key":"cit0084","doi-asserted-by":"publisher","DOI":"10.3389\/fncel.2018.00188"},{"key":"cit0085","doi-asserted-by":"publisher","DOI":"10.1016\/j.freeradbiomed.2009.07.035"},{"key":"cit0086","doi-asserted-by":"publisher","DOI":"10.1111\/acel.12216"},{"key":"cit0087","doi-asserted-by":"publisher","DOI":"10.1016\/j.cotox.2016.10.002"},{"key":"cit0088","doi-asserted-by":"publisher","DOI":"10.1093\/toxsci\/kfr183"},{"key":"cit0089","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0153574"},{"key":"cit0090","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.660"},{"key":"cit0091","doi-asserted-by":"publisher","DOI":"10.2147\/DDDT.S193889"},{"key":"cit0092","doi-asserted-by":"publisher","DOI":"10.1042\/NS20180060"},{"key":"cit0093","doi-asserted-by":"publisher","DOI":"10.2147\/DNND.S180027"},{"key":"cit0094","doi-asserted-by":"publisher","DOI":"10.2139\/ssrn.3576913"},{"key":"cit0095","doi-asserted-by":"publisher","DOI":"10.1101\/2022.08.12.22278684"},{"key":"cit0096","doi-asserted-by":"publisher","DOI":"10.1016\/j.phrs.2020.104994"},{"key":"cit0097","doi-asserted-by":"publisher","DOI":"10.31525\/ct1-nct04008186"},{"key":"cit0098","unstructured":"CYTOCHROME P450 DRUG INTERACTION TABLE - Drug Interactions [Internet]. 2022 [cited 2022 Nov 8]. Available from: https:\/\/drug-interactions.medicine.iu.edu\/MainTable.aspx."},{"key":"cit0099","doi-asserted-by":"publisher","DOI":"10.1177\/0883073816643408"},{"key":"cit0100","unstructured":"Reata Pharmaceuticals Inc. Investors - News [Internet]. [cited 2022 Nov 9]. Available from: https:\/\/www.reatapharma.com\/investors\/news\/default.aspx."}],"container-title":["Expert Opinion on Investigational Drugs"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.tandfonline.com\/doi\/pdf\/10.1080\/13543784.2023.2173063","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,2,17]],"date-time":"2023-02-17T07:59:56Z","timestamp":1676620796000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.tandfonline.com\/doi\/full\/10.1080\/13543784.2023.2173063"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,1,2]]},"references-count":100,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2023,1,2]]}},"alternative-id":["10.1080\/13543784.2023.2173063"],"URL":"https:\/\/doi.org\/10.1080\/13543784.2023.2173063","relation":{},"ISSN":["1354-3784","1744-7658"],"issn-type":[{"value":"1354-3784","type":"print"},{"value":"1744-7658","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,1,2]]},"assertion":[{"value":"The publishing and review policy for this title is described in its Aims & Scope.","order":1,"name":"peerreview_statement","label":"Peer Review Statement"},{"value":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ieid20","URL":"http:\/\/www.tandfonline.com\/action\/journalInformation?show=aimsScope&journalCode=ieid20","order":2,"name":"aims_and_scope_url","label":"Aim & Scope"},{"value":"2022-11-09","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-01-23","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-02-08","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}