Synonyms | EPI-743; EPI 743; Vatiquinone; alpha-Tocotrienol quinone.;2-((R,6E,10E)-3-hydroxy-3,7,11,15-tetramethylhexadeca-6,10,14-trien-1-yl)-3,5,6-trimethylcyclohexa-2,5-diene-1,4-dione
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Reference | 1: Enns GM, Kinsman SL, Perlman SL, Spicer KM, Abdenur JE, Cohen BH, Amagata A,
Barnes A, Kheifets V, Shrader WD, Thoolen M, Blankenberg F, Miller G. Initial
experience in the treatment of inherited mitochondrial disease with EPI-743. Mol
Genet Metab. 2012 Jan;105(1):91-102. doi: 10.1016/j.ymgme.2011.10.009. Epub 2011
Oct 21. PubMed PMID: 22115768.<br />
2: Pastore A, Petrillo S, Tozzi G, Carrozzo R, Martinelli D, Dionisi-Vici C, Di
Giovamberardino G, Ceravolo F, Klein MB, Miller G, Enns GM, Bertini E, Piemonte
F. Glutathione: a redox signature in monitoring EPI-743 therapy in children with
mitochondrial encephalomyopathies. Mol Genet Metab. 2013 Jun;109(2):208-14. doi:
10.1016/j.ymgme.2013.03.011. Epub 2013 Mar 24. PubMed PMID: 23583222.<br />
3: Martinelli D, Catteruccia M, Piemonte F, Pastore A, Tozzi G, Dionisi-Vici C,
Pontrelli G, Corsetti T, Livadiotti S, Kheifets V, Hinman A, Shrader WD, Thoolen
M, Klein MB, Bertini E, Miller G. EPI-743 reverses the progression of the
pediatric mitochondrial disease–genetically defined Leigh Syndrome. Mol Genet
Metab. 2012 Nov;107(3):383-8. doi: 10.1016/j.ymgme.2012.09.007. Epub 2012 Sep 10.
PubMed PMID: 23010433.<br />
4: Chicani CF, Chu ER, Miller G, Kelman SE, Sadun AA. Comparing EPI-743 treatment
in siblings with Leber/’s hereditary optic neuropathy mt14484 mutation. Can J
Ophthalmol. 2013 Oct;48(5):e130-3. doi: 10.1016/j.jcjo.2013.05.011. PubMed PMID:
24093206.<br />
5: Sadun AA, Chicani CF, Ross-Cisneros FN, Barboni P, Thoolen M, Shrader WD,
Kubis K, Carelli V, Miller G. Effect of EPI-743 on the clinical course of the
mitochondrial disease Leber hereditary optic neuropathy. Arch Neurol. 2012
Mar;69(3):331-8. doi: 10.1001/archneurol.2011.2972. PubMed PMID: 22410442. <br />
6: Blankenberg FG, Kinsman SL, Cohen BH, Goris ML, Spicer KM, Perlman SL, Krane
EJ, Kheifets V, Thoolen M, Miller G, Enns GM. Brain uptake of Tc99m-HMPAO
correlates with clinical response to the novel redox modulating agent EPI-743 in
patients with mitochondrial disease. Mol Genet Metab. 2012 Dec;107(4):690-9. doi:
10.1016/j.ymgme.2012.09.023. Epub 2012 Sep 28. PubMed PMID: 23084792.
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