Developments in the Treatment of Leber Hereditary Optic Neuropathy.
Chen BS, Yu-Wai-Man P, Newman NJ.
Curr Neurol Neurosci Rep. 2022 Dec;22(12):881-892. doi: 10.1007/s11910-022-01246-y. Epub 2022 Nov 21.
Therapeutic benefit of idebenone in patients with Leber hereditary optic neuropathy: The LEROS nonrandomized controlled trial.
Yu-Wai-Man P, Carelli V, Newman NJ, Silva MJ, Linden A, Van Stavern G, Szaflik JP, Banik R, Lubiński W, Pemp B, Liao YJ, Subramanian PS, Misiuk-Hojło M, Newman S, Castillo L, Kocięcki J, Levin MH, Muñoz-Negrete FJ, Yagan A, Cherninkova S, Katz D, Meunier A, Votruba M, Korwin M, Dziedziak J, Jurkutė N, Harvey JP, La Morgia C, Priglinger C, Llòria X, Tomasso L, Klopstock T; LEROS Study Group.
Cell Rep Med. 2024 Mar 19;5(3):101437. doi: 10.1016/j.xcrm.2024.101437. Epub 2024 Feb 29.
Mitochondrial Disorders.
Klopstock T, Priglinger C, Yilmaz A, Kornblum C, Distelmaier F, Prokisch H.
Dtsch Arztebl Int. 2021 Nov 5;118(44):741-748. doi: 10.3238/arztebl.m2021.0251.
Randomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy.
Newman NJ, Yu-Wai-Man P, Subramanian PS, Moster ML, Wang AG, Donahue SP, Leroy BP, Carelli V, Biousse V, Vignal-Clermont C, Sergott RC, Sadun AA, Rebolleda Fernández G, Chwalisz BK, Banik R, Bazin F, Roux M, Cox ED, Taiel M, Sahel JA; LHON REFLECT Study Group.
Brain. 2023 Apr 19;146(4):1328-1341. doi: 10.1093/brain/awac421.
Leber hereditary optic neuropathy: new and emerging therapies.
Davila-Siliezar P, Carter M, Milea D, Lee AG.
Curr Opin Ophthalmol. 2022 Nov 1;33(6):574-578. doi: 10.1097/ICU.0000000000000891. Epub 2022 Aug 24.
Leber hereditary optic neuropathy.
Yu-Wai-Man P, Turnbull DM, Chinnery PF.
J Med Genet. 2002 Mar;39(3):162-9. doi: 10.1136/jmg.39.3.162.
Recognizing Leber's Hereditary Optic Neuropathy to avoid delayed diagnosis and misdiagnosis.
La Morgia C, Cascavilla ML, De Negri AM, Romano M, Canalini F, Rossi S, Centonze D, Filippi M.
Front Neurol. 2024 Sep 19;15:1466275. doi: 10.3389/fneur.2024.1466275. eCollection 2024.
Leber's Hereditary Optic Neuropathy: Novel Views and Persisting Challenges.
Jancic J, Samardzic J, Stojanovic S, Stojanovic A, Milanovic AM, Nikolic B, Ivancevic N, Kostic V.
CNS Neurol Disord Drug Targets. 2017;16(8):927-935. doi: 10.2174/1871527316666170724172455.
Gene Therapy for Leber's Hereditary Optic Neuropathy: Time to Include a True Placebo Arm?
Wang Q, Milea D, Lee AG.
Am J Ophthalmol. 2022 Sep;241:A11-A12. doi: 10.1016/j.ajo.2022.05.018. Epub 2022 May 27.
Primary mitochondrial diseases.
Pizzamiglio C, Hanna MG, Pitceathly RDS.
Handb Clin Neurol. 2024;204:53-76. doi: 10.1016/B978-0-323-99209-1.00004-1.
