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A randomized placebo controlled study published in the journal Brain showed idebenone to be an effective treatment for LHON. Idebenone is a synthetic benzoquinone, co-enzyme Q10 like substance, potent antioxidant and inhibitor of lipid peroxidation, which also interacts with the mitochondrial electron transport chain and facilitates mitochondrial electron flux through by-passing complex I. It was shown in the study to be well tolerated and have modest effectiveness in about half of LHON patients treated. The result confirms retrospective treatment data now published. People treated early in onset and for prolonged periods were likely to benefit most. This has been confirmed in real world clinical experience.
Use of Idebenone for the Treatment of Leber’s Hereditary Optic Neuropathy: Review of the Evidence Claudia B. Catarino MD, PhD, Thomas Klopstock, MD. Journal of Inborn Errors of Metabolism and Screening. Sage Journals. First Published September 18, 2017 Review Article. https://journals.sagepub.com/doi/full/10.1177/2326409817731112
Catarino, Claudia B. MD, PhD; von Livonius, Bettina MD; Priglinger, Claudia MD; Banik, Rudrani MD; Matloob, Selma MBChB, MRCOphth; Tamhankar, Madhura A. MD; Castillo, Lorena MD; Friedburg, Christoph MD, FEBO; Halfpenny, Christopher A. MBBS, PhD; Lincoln, John A. MD, PhD; Traber, Ghislaine L. MD; Acaroglu, Gölge MD; Black, Graeme C. M. DPhil, FRCOphth; Doncel, Carlos MD; Fraser, Clare L. MBBS, MMed; Jakubaszko, Joanna MD, PhD; Landau, Klara MD, FEBO; Langenegger, Stefan J. MD, FEBO; Muñoz-Negrete, Francisco J. MD, PhD; Newman, Nancy J. MD; Poulton, Joanna DM, MRCPCH; Scoppettuolo, Elisabetta MBBS, FRCOphth; Subramanian, Prem MD, PhD; Toosy, Ahmed T. MBBS, FRCP; Vidal, Mariona MD; Vincent, Andrea L. MBChB, MD; Votruba, Marcela BM BCh, FRCOphth; Zarowski, Marcin MD, PhD; Zermansky, Adam MBChB, FRCP; Lob, Felice MD; Rudolph, Günther MD; Mikazans, Oskars MA; Silva, Magda; Llòria, Xavier MD; Metz, Günther PhD; Klopstock, Thomas MD Real-World Clinical Experience With Idebenone in the Treatment of Leber Hereditary Optic Neuropathy, Journal of Neuro-Ophthalmology: September 22, 2020 - Volume Publish Ahead of Print - Issue - doi: 10.1097/WNO.0000000000001023 https://doi.org/10.1097/WNO.0000000000001023
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