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

39 clinical trials for this condition and its sub-types, 18 tagged with Leber hereditary optic neuropathy itself.

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Where it sits in the disease tree

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↑ Familial dilated cardiomyopathy (60) ↑ Mitochondrial oxidative phosphorylation disorder (58) ↑ Hereditary optic neuropathy (30) ↑ Hereditary optic atrophy (23)

Sub-types of Leber hereditary optic neuropathy

Leber optic atrophy and dystonia 0 trials

Including sub-types (39) Tagged with Leber hereditary optic neuropathy (18)

Trials to join now! 7 Not yet recruiting 1 Not yet finished but already full! 4 Completed 6

Could vitamin B3 save sight in rare genetic blindness?

Disease control Not yet recruiting

This early study tests whether high-dose vitamin B3 (nicotinamide) can help preserve or improve vision in people with Leber's hereditary optic neuropathy (LHON), a rare genetic disease that causes sudden vision loss. Researchers will give 13 participants 2 grams of vitamin B3 dai…

Phase 1 • Sponsor: University Hospital, Angers • Aim: Disease control

Last updated Jun 27, 2026 08:05 UTC

Leber hereditary optic neuropathy

Where it sits in the disease tree

Could vitamin B3 save sight in rare genetic blindness?