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Leber hereditary optic neuropathy
MONDO:0010788Leber'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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Sub-types of Leber hereditary optic neuropathy
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Leber optic atrophy and dystonia 0 trials
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Desperate hope: gene therapy tested in one patient with rare blindness
Disease control Expanded access (ended)This expanded access program gave a single patient with Leber Hereditary Optic Neuropathy (a genetic cause of vision loss) an experimental gene therapy called GS010. The treatment was injected into both eyes to test safety. Only one person was involved, so the results are very li…
Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Gene therapy aims to restore sight in rare blindness condition
Disease control OngoingThis phase 3 trial tests a gene therapy called NR082 for Leber's hereditary optic neuropathy (LHON), a genetic condition that causes rapid vision loss. About 95 people aged 12 to 75 with a specific ND4 mutation will receive a single injection of the therapy or a sham procedure. T…
Phase 3 • Sponsor: Wuhan Neurophth Biotechnology Limited Company • Aim: Disease control
Last updated Jun 27, 2026 09:05 UTC
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Massive genetic study aims to unlock secrets of rare metabolic diseases
Knowledge-focused By invitation onlyThis study will collect and analyze genetic data from 1000 people with suspected inherited metabolic diseases, including conditions like epilepsy and mitochondrial disorders. Researchers at Karolinska University Hospital aim to improve diagnosis by using advanced genetic testing …
Sponsor: Region Stockholm • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:38 UTC
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Massive gene hunt launched for mysterious mitochondrial diseases
Knowledge-focused By invitation onlyThis study aims to discover new genetic mutations that cause mitochondrial disorders by analyzing tissue samples from up to 6,900 participants. It includes people with suspected or known mitochondrial diseases, such as MELAS or Leigh's Disease, who lack a genetic diagnosis. The r…
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:09 UTC