Title |
International Consensus Statement on the Clinical and Therapeutic Management of Leber Hereditary Optic Neuropathy
|
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Published in |
Journal of Neuro-Ophthalmology, December 2017
|
DOI | 10.1097/wno.0000000000000570 |
Pubmed ID | |
Authors |
Valerio Carelli, Michele Carbonelli, Irenaeus F. de Coo, Aki Kawasaki, Thomas Klopstock, Wolf A. Lagrèze, Chiara La Morgia, Nancy J. Newman, Christophe Orssaud, Jan Willem R. Pott, Alfredo A. Sadun, Judith van Everdingen, Catherine Vignal-Clermont, Marcela Votruba, Patrick Yu-Wai-Man, Piero Barboni |
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. |
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Geographical breakdown
Country | Count | As % |
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Spain | 1 | 25% |
Australia | 1 | 25% |
United States | 1 | 25% |
Unknown | 1 | 25% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 2 | 50% |
Science communicators (journalists, bloggers, editors) | 1 | 25% |
Scientists | 1 | 25% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 121 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
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Researcher | 19 | 16% |
Other | 18 | 15% |
Student > Bachelor | 11 | 9% |
Student > Ph. D. Student | 10 | 8% |
Student > Master | 10 | 8% |
Other | 23 | 19% |
Unknown | 30 | 25% |
Readers by discipline | Count | As % |
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Medicine and Dentistry | 44 | 36% |
Biochemistry, Genetics and Molecular Biology | 12 | 10% |
Neuroscience | 8 | 7% |
Pharmacology, Toxicology and Pharmaceutical Science | 7 | 6% |
Agricultural and Biological Sciences | 5 | 4% |
Other | 11 | 9% |
Unknown | 34 | 28% |