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Title: | Treatment of Leber’s hereditary optic neuropathy : an overview of recent developments |
Authors: | Zuccarelli, Marta Vella Szijj, Janis Serracino-Inglott, Anthony Borg, John-Joseph |
Keywords: | Genetics Glaucoma -- Diagnosis Neuroophthalmology -- Case studies Neuropathy Pediatrics -- Case studies Pharmacology -- Case studies |
Issue Date: | 2020 |
Publisher: | Sage Publications Ltd. |
Citation: | Zuccarelli, M., Vella-Szijj, J., Serracino-Inglott, A., & Borg, J. J. (2020). Treatment of Leber’s hereditary optic neuropathy: An overview of recent developments. European Journal of Ophthalmology, 1120672120936592. |
Abstract: | Leber’s hereditary optic neuropathy (LHON) is a rare, maternally-inherited optic neuropathy caused by mitochondrial DNA point mutations and which can cause blindness. Currently, Raxone (idebenone) is the only available medicinal product authorised to treat LHON within the European Union and LHON remains an unmet medical need. The aim of this article was to summarise interventional clinical trials published over the past 5 years (between 2014 and 2019) with the primary purpose of treating LHON. Therapeutic approaches discussed include modulating agents of the mitochondrial electron transport chain such as Raxone, cysteamine bitartrate and KH176, inhibitors of apoptosis such as elamipretide, gene therapy medicinal products such as GS010 and scAAV2P1ND4 and retinal tissue regeneration medicinal products such as bone marrow-derived stem cells. |
URI: | https://www.um.edu.mt/library/oar/handle/123456789/60684 |
Appears in Collections: | Scholarly Works - FacM&SPha |
Files in This Item:
File | Description | Size | Format | |
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Treatment_of_Lebers_hereditary_optic_neuropathy.pdf | 1.03 MB | Adobe PDF | View/Open |
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