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International consensus statement on the clinical and therapeutic management of Leber's Hereditary Optic Neuropathy.

Carelli, V, Carbonelli, M, de Coo, I, Kawasaki, A, Klopstock, T, Lagrèze, W, La Morgia, C, Newman, N. J., Orssaud, C, Pott, J. W., Sadun, A, Everdingen, J, Vignal-Clermont, C, Votruba, Marcela, Yu-Wai-Man, P and Barboni, P 2017. International consensus statement on the clinical and therapeutic management of Leber's Hereditary Optic Neuropathy. Journal of Neuro-Ophthalmology 37 (4) , pp. 371-381. 10.1097/WNO.0000000000000570

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Abstract

Leber hereditary optic neuropathy (LHON) is currently estimated as the most frequent mitochondrial disease (1 in 27,000-45,000). Its molecular pathogenesis and natural history is now fairly well understood. LHON also is the first mitochondrial disease for which a treatment has been approved (idebenone-Raxone, Santhera Pharmaceuticals) by the European Medicine Agency, under exceptional circumstances because of the rarity and severity of the disease. However, what remains unclear includes the optimal target population, timing, dose, and frequency of administration of idebenone in LHON due to lack of accepted definitions, criteria, and general guidelines for the clinical management of LHON. To address these issues, a consensus conference with a panel of experts from Europe and North America was held in Milan, Italy, in 2016. The intent was to provide expert consensus statements for the clinical and therapeutic management of LHON based on the currently available evidence. We report the conclusions of this conference, providing the guidelines for clinical and therapeutic management of LHON.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Optometry and Vision Sciences
Publisher: Lippincott, Williams & Wilkins
ISSN: 1070-8022
Date of First Compliant Deposit: 23 October 2017
Date of Acceptance: 17 August 2017
Last Modified: 01 Dec 2018 02:30
URI: http://orca.cf.ac.uk/id/eprint/105811

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