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
-
Leber hereditary optic neuropathy 18 trials
1 sub-type
- Leber optic atrophy and dystonia 0 trials
-
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
-
Optic atrophy 6 1 trial
-
1 sub-type
- Optic atrophy 9 0 trials
-
Optic atrophy 11 0 trials
-
Optic atrophy 12 0 trials
-
Optic atrophy 14 0 trials
-
Optic atrophy 15 0 trials
-
Optic atrophy 16 0 trials
-
Optic atrophy 2 0 trials
-
Optic atrophy 4 0 trials
Most studied deeper sub-types
-
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
-
Gene therapy shows promise in Long-Term study for rare blindness
Disease control CompletedThis study followed 62 people with Leber Hereditary Optic Neuropathy (LHON), a rare inherited eye disease that causes vision loss, for up to 5 years after they received a single gene therapy treatment called GS010. The goal was to see if the treatment remained safe and if any vis…
Phase 3 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
-
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
-
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
-
Gene therapy for inherited blindness passes early safety check
Disease control CompletedThis study tested a gene therapy called GS010 in 19 adults with Leber Hereditary Optic Neuropathy (LHON), a rare inherited eye disease that causes rapid vision loss. The main goal was to see if the treatment is safe and tolerable at different doses. The therapy uses a harmless vi…
Phase 1/2 • Sponsor: GenSight Biologics • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
-
New study tracks daily life impact of rare eye disease
Knowledge-focused CompletedThis study looked at 44 people with a rare inherited eye condition called Leber Hereditary Optic Neuropathy (LHON) that causes vision loss. Researchers reviewed medical records and asked participants about their vision, health, and quality of life. The goal was to better understa…
Sponsor: GenSight Biologics • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:09 UTC