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Leber congenital amaurosis 1

MONDO:0008764

Any Leber congenital amaurosis in which the cause of the disease is a mutation in the GUCY2D gene.

Also known as: GUCY2D Leber congenital amaurosis, LCA1, Leber congenital amaurosis 1, Leber congenital amaurosis caused by mutation in GUCY2D, Leber congenital amaurosis type 1, CRB, LCA, amaurosis congenita of Leber 1

26 clinical trials for this condition and its sub-types.

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Broader categories

Disease (717) Nervous system disorder (243) Hereditary disease (188) Eye disorder (104) Retinal disorder (89) Inherited retinal dystrophy (41) Retinal degeneration (41) Perceptual disorders (22) Human disease (15) Leber congenital amaurosis (9)
Trials to join now! 11 Not yet recruiting 3 Not yet finished but already full! 1 Completed 9 Terminated 2
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  • 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

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