Hereditary optic atrophy

MONDO:0043878

A 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.

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Where it sits in the disease tree

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Part of

↑ Inherited neurodegenerative disorder (808) ↑ Primary optic atrophy (23)

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
ACO2-related optic atrophy with or without extraocular features 0 trials
1 sub-type
- Optic atrophy 9 0 trials
Autosomal recessive optic atrophy, OPA7 type 0 trials
Optic atrophy 10 with or without ataxia, intellectual disability, and seizures 0 trials
Optic atrophy 11 0 trials
Optic atrophy 12 0 trials
Optic atrophy 13 with retinal and foveal abnormalities 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
Osteogenesis imperfecta-retinopathy-seizures-intellectual disability syndrome 0 trials

Most studied deeper sub-types

Cerebellar ataxia-areflexia-pes cavus-optic atrophy-sensorineural hearing loss syndrome (0) Optic atrophy 8 (0) Optic atrophy, hearing loss, and peripheral neuropathy, autosomal dominant (0) Optic atrophy with or without deafness, ophthalmoplegia, myopathy, ataxia, and neuropathy (0)

Including sub-types (26) Tagged with Hereditary optic atrophy (6)

Trials to join now! 1 Not yet recruiting 1 Not yet finished but already full! 1 Completed 3

New eye drug hopes to slow inherited blindness

Disease control Recruiting now

This early-stage study tests a new medicine called PYC-001, given as an injection into the eye, for people with a rare genetic eye disease (autosomal dominant optic atrophy) caused by a change in the OPA1 gene. The main goal is to check the safety of different doses and schedules…

Phase 1/2 • Sponsor: PYC Therapeutics • Aim: Disease control

Last updated Jul 01, 2026 00:00 UTC

Hereditary optic atrophy

Where it sits in the disease tree

Most studied deeper sub-types

New eye drug hopes to slow inherited blindness