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GUCY2D retinopathy

MONDO:0100454

Any inherited retinal dystrophy in which the cause of the disease is a mutation in the GUCY2D gene.

Also known as: retinopathy caused by mutation in GUCY2D

26 clinical trials for this condition and its sub-types, 0 tagged with GUCY2D retinopathy itself.

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Where it sits in the disease tree

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Part of

↑ Inherited retinal dystrophy (510)

Sub-types of GUCY2D retinopathy

Cone-rod dystrophy 6 1 trial
GUCY2D-related recessive retinopathy 0 trials · 1 incl. sub-types
2 sub-types
- Leber congenital amaurosis 1 1 trial
- Night blindness, congenital stationary, type1i 0 trials
GUCY2D-related dominant retinopathy 0 trials
1 sub-type
- Choroidal dystrophy, central areolar, 1 0 trials

Including sub-types (26) Tagged with GUCY2D retinopathy (0)

Trials to join now! 11 Not yet recruiting 3 Not yet finished but already full! 1 Completed 9 Terminated 2

Can a single injection restore sight in a rare childhood blindness?

Cure Ongoing

This trial tests a gene therapy called ATSN-101, given as a one-time injection under the retina, for people with Leber congenital amaurosis caused by GUCY2D gene mutations. The goal is to see if the treatment is safe and can improve vision. Participants receive the therapy in one…

Phase 1/2 • Sponsor: Atsena Therapeutics Inc. • Aim: Cure

Last updated Aug 05, 2026 00:00 UTC