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Leber hereditary optic neuropathy

MONDO:0010788

Leber's hereditary optic neuropathy (LHON) is a mitochondrial neurodegenerative disease affecting the optic nerve and often characterized by sudden vision loss in young adult carriers.

Also known as: LHON, Leber Hereditary optic atrophy, Leber hereditary optic neuropathy, Leber optic atrophy, Leber’s disease, optic atrophy, Leber type

38 clinical trials for this condition and its sub-types.

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Sub-types

Leber optic atrophy and dystonia (0)

Broader categories

Cardiovascular disorder (1051) Disease (680) Heart disorder (300) Metabolic disease (233) Nervous system disorder (231) Musculoskeletal system disorder (207) Hereditary disease (176) Neurodegenerative disease (171) Cardiomyopathy (144) Central nervous system disorder (107)

Trials to join now! 17 Not yet recruiting 2 Not yet finished but already full! 10 Completed 8 Terminated 1

New drug aims to tame hard-to-control seizures in rare mitochondrial disorders

Disease control Terminated

This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …

Phase: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 12:03 UTC