Treatment of Leber’s hereditary optic neuropathy: An overview of recent developments

Treatment of Leber’s hereditary optic neuropathy: An overview of recent developments

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 rem...

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Veröffentlicht in:European Journal of Ophthalmology 2020-11, Vol.30 (6), p.1220-1227
Hauptverfasser: Zuccarelli, Marta, Vella-Szijj, Janis, Serracino-Inglott, Anthony, Borg, John-Joseph
Format: Artikel
Sprache:eng
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Disease Management
DNA, Mitochondrial - genetics
Humans
Optic Atrophy, Hereditary, Leber - genetics
Optic Atrophy, Hereditary, Leber - therapy
Retina - pathology
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container_end_page 1227
container_issue 6
container_start_page 1220
container_title European Journal of Ophthalmology
container_volume 30
creator Zuccarelli, Marta
Vella-Szijj, Janis
Serracino-Inglott, Anthony
Borg, John-Joseph
description 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.
doi_str_mv 10.1177/1120672120936592
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subjects Disease Management
DNA, Mitochondrial - genetics
Humans
Optic Atrophy, Hereditary, Leber - genetics
Optic Atrophy, Hereditary, Leber - therapy
Retina - pathology
title Treatment of Leber’s hereditary optic neuropathy: An overview of recent developments
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