{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T15:57:21Z","timestamp":1787068641431,"version":"build-2736575974"},"reference-count":20,"publisher":"SAGE Publications","issue":"6","license":[{"start":{"date-parts":[[2020,6,19]],"date-time":"2020-06-19T00:00:00Z","timestamp":1592524800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["European Journal of Ophthalmology"],"published-print":{"date-parts":[[2020,11]]},"abstract":"<jats:p>Leber\u2019s hereditary optic neuropathy (LHON) is a rare, maternally-inherited optic neuropathy caused by mitochondrial DNA point mutations and which can cause blindness. Currently, Raxone (idebenone) is the only available medicinal product authorised to treat LHON within the European Union and LHON remains an unmet medical need. The aim of this article was to summarise interventional clinical trials published over the past 5\u2009years (between 2014 and 2019) with the primary purpose of treating LHON. Therapeutic approaches discussed include modulating agents of the mitochondrial electron transport chain such as Raxone, cysteamine bitartrate and KH176, inhibitors of apoptosis such as elamipretide, gene therapy medicinal products such as GS010 and scAAV2P1ND4 and retinal tissue regeneration medicinal products such as bone marrow-derived stem cells.<\/jats:p>","DOI":"10.1177\/1120672120936592","type":"journal-article","created":{"date-parts":[[2020,6,19]],"date-time":"2020-06-19T05:54:01Z","timestamp":1592546041000},"page":"1220-1227","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":28,"title":["Treatment of Leber\u2019s hereditary optic neuropathy: An overview of recent developments"],"prefix":"10.1177","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7577-3807","authenticated-orcid":false,"given":"Marta","family":"Zuccarelli","sequence":"first","affiliation":[{"name":"Medicines Authority, San \u0120wann, Malta"},{"name":"Department of Pharmacy, Faculty of Medicine and Surgery, University of Malta, Msida, Malta"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Janis","family":"Vella-Szijj","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, Faculty of Medicine and Surgery, University of Malta, Msida, Malta"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anthony","family":"Serracino-Inglott","sequence":"additional","affiliation":[{"name":"Medicines Authority, San \u0120wann, Malta"},{"name":"Department of Pharmacy, Faculty of Medicine and Surgery, University of Malta, Msida, Malta"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John-Joseph","family":"Borg","sequence":"additional","affiliation":[{"name":"Medicines Authority, San \u0120wann, Malta"},{"name":"Department of Biology, University of Tor Vergata, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2020,6,19]]},"reference":[{"key":"bibr1-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1016\/j.preteyeres.2010.11.002"},{"key":"bibr2-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1038\/srep21587."},{"key":"bibr3-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1177\/2326409817731112."},{"key":"bibr4-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1016\/j.mito.2017.01.004"},{"key":"bibr5-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1097\/ICU.0000000000000410"},{"key":"bibr6-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1016\/j.phrs.2018.10.014"},{"issue":"9","key":"bibr7-1120672120936592","doi-asserted-by":"crossref","DOI":"10.1093\/brain\/awr180","volume":"134","author":"Carelli V","year":"2011","journal-title":"Brain"},{"key":"bibr8-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1001\/jamaophthalmol.2013.7630"},{"key":"bibr9-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1242\/dmm.020248"},{"key":"bibr10-1120672120936592","doi-asserted-by":"publisher","DOI":"10.3797\/scipharm.1402-14"},{"key":"bibr11-1120672120936592","unstructured":"European Medicines Agency. Raxone: EPAR \u2013 public assessment report, https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/raxone (2015, accessed 22 October 2019)."},{"key":"bibr12-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbadis.2011.02.010"},{"key":"bibr13-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-017-0715-0"},{"key":"bibr14-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1155\/2015\/847985"},{"key":"bibr15-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1016\/j.plipres.2014.04.001"},{"key":"bibr16-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacbts.2018.12.005"},{"key":"bibr17-1120672120936592","unstructured":"GenSight Biologics. GenSight Biologics reports positive follow-up results at week 72 of the RESCUE phase III clinical trial of GS010 in Leber hereditary optic neuropathy (LHON), https:\/\/www.gensight-biologics.com\/2019\/04\/17\/gensight-biologics-reports-positive-follow-up-results-at-week-72-of-the-rescue-phase-iii-clinical-trial-of-gs010-in-leber-hereditary-optic-neuropathy-lhon\/ (2019, accessed 22 October 2019)."},{"key":"bibr18-1120672120936592","unstructured":"GenSight Biologics. GenSight Biologics reports positive 96-week data from REVERSE phase III clinical trial of GS010 for the treatment of Leber hereditary optic neuropathy (LHON), https:\/\/www.gensight-biologics.com\/2019\/05\/15\/gensight-biologics-reports-positive-96-week-data-from-reverse-phase-iii-clinical-trial-of-gs010-for-the-treatment-of-leber-hereditary-optic-neuropathy-lhon\/ (2019, accessed 22 October 2019)."},{"key":"bibr19-1120672120936592","doi-asserted-by":"publisher","DOI":"10.1016\/j.ophtha.2017.05.016"},{"key":"bibr20-1120672120936592","doi-asserted-by":"publisher","DOI":"10.4103\/1673-5374.193251"}],"container-title":["European Journal of Ophthalmology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/1120672120936592","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/1120672120936592","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/1120672120936592","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,4,28]],"date-time":"2026-04-28T19:45:52Z","timestamp":1777405552000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/1120672120936592"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,6,19]]},"references-count":20,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2020,11]]}},"alternative-id":["10.1177\/1120672120936592"],"URL":"https:\/\/doi.org\/10.1177\/1120672120936592","relation":{},"ISSN":["1120-6721","1724-6016"],"issn-type":[{"value":"1120-6721","type":"print"},{"value":"1724-6016","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,6,19]]}}}