Article
Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.
Cell reports. Medicine - 19 Mar 2024
Yu-Wai-Man Patrick, Carelli Valerio, Newman Nancy J, Silva Magda Joana, Linden Aki, Van Stavern Gregory, Szaflik Jacek P, Banik Rudrani, Lubiński Wojciech, Pemp Berthold, Liao Yaping Joyce, Subramanian Prem S, Misiuk-Hojło Marta, Newman Steven, Castillo Lorena, Kocięcki Jarosław, Levin Marc H, Muñoz-Negrete Francisco Jose, Yagan Ali, Cherninkova Sylvia, Katz David, Meunier Audrey, Votruba Marcela, Korwin Magdalena, Dziedziak Jacek, Jurkutė Neringa, Harvey Joshua P, La Morgia Chiara, Priglinger Claudia, Llòria Xavier, Tomasso Livia, Klopstock Thomas
Abstract excerpt
Leber hereditary optic neuropathy (LHON) is a mitochondrial disease leading to rapid and severe bilateral vision loss. Idebenone has been shown to be effective in stabilizing and restoring vision in patients treated within 1 year of onset of vision loss. The open-label, international, multicenter, natural history-controlled LEROS study (ClinicalTrials.gov NCT02774005) assesses the efficacy and safety of idebenone...
Topics
- Humans
- Optic Atrophy, Hereditary, Leber
- Antioxidants
- Ubiquinone
- Mutation