EPI-743 reverses the progression of the pediatric mitochondrial disease--genetically defined Leigh Syndrome.
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.
Martinelli D, et al. Among authors: dionisi vici c.
Mol Genet Metab. 2012 Nov;107(3):383-8. doi: 10.1016/j.ymgme.2012.09.007. Epub 2012 Sep 10.
Mol Genet Metab. 2012.
PMID: 23010433
Clinical Trial.