Hereditary optic atrophy
MONDO:0043878A family of inherited disorders characterized by progressive loss of vision secondary to death of the retinal ganglion cell axons that comprise the optic nerve.
Also known as: hereditary optic atrophy, Atrophies, hereditary optic, atrophy, hereditary optic, hereditary optic Atrophies, optic atrophy, hereditary
26 clinical trials for this condition and its sub-types, 6 tagged with Hereditary optic atrophy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Hereditary optic atrophy
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Leber hereditary optic neuropathy 18 trials
1 sub-type
- Leber optic atrophy and dystonia 0 trials
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Autosomal dominant optic atrophy 6 trials
6 sub-types
- Autosomal dominant optic atrophy, classic form 3 trials
- Al Gazali Khidr Prem Chandran syndrome 0 trials
- Autosomal dominant optic atrophy and peripheral neuropathy 0 trials
- Autosomal dominant optic atrophy plus syndrome 0 trials Sub-types →
- Optic atrophy 3 0 trials
- Optic atrophy 5 0 trials
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Optic atrophy 6 1 trial
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1 sub-type
- Optic atrophy 9 0 trials
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Optic atrophy 11 0 trials
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Optic atrophy 12 0 trials
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Optic atrophy 14 0 trials
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Optic atrophy 15 0 trials
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Optic atrophy 16 0 trials
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Optic atrophy 2 0 trials
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Optic atrophy 4 0 trials
Most studied deeper sub-types
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Gene therapy shows promise for rare eye disease that causes blindness
Disease control CompletedThis Phase 3 trial tested a gene therapy called GS010 in 98 people with Leber hereditary optic neuropathy (LHON), a genetic condition that leads to rapid vision loss. Participants received injections of the therapy or a placebo into both eyes. The study measured changes in vision…
Phase 3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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Gene therapy injection shows promise for rare eye disease
Disease control CompletedThis phase 3 trial tested a single injection of GS010 gene therapy in 39 people with Leber Hereditary Optic Neuropathy (LHON) who had vision loss for 6 months or less. The treatment aims to improve vision by delivering a working copy of the ND4 gene to the eye. Results measured c…
Phase 3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Gene therapy shows promise for rare eye disease causing blindness
Disease control CompletedThis study tested a one-time gene therapy injection, GS010, in 37 adults with Leber Hereditary Optic Neuropathy (LHON), a genetic condition that causes rapid vision loss. Participants had vision loss for 6 to 12 months before treatment. The goal was to see if the therapy could im…
Phase 3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC