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Leber congenital amaurosis 10

MONDO:0012723

Any Leber congenital amaurosis in which the cause of the disease is a mutation in the CEP290 gene.

Also known as: CEP290 Leber congenital amaurosis, LCA10, Leber congenital amaurosis 10, Leber congenital amaurosis caused by mutation in CEP290, Leber congenital amaurosis type 10, amaurosis congenita of Leber, type 10

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

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Broader categories

Disease (680) Nervous system disorder (231) Hereditary disease (176) Eye disorder (102) Retinal disorder (85) Inherited retinal dystrophy (40) Retinal degeneration (40) Perceptual disorders (22) Human disease (14) Leber congenital amaurosis (9)
Trials to join now! 13 Not yet recruiting 3 Not yet finished but already full! 1 Completed 8 Terminated 2
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  • Can a single eye injection restore sight in genetic blindness?

    ⭐️ CURE ⭐️ Ongoing

    This trial tests an experimental gene therapy called OCU400 in people with retinitis pigmentosa or Leber congenital amaurosis, which are inherited conditions that cause progressive vision loss and can lead to blindness. The therapy is given as a single injection into the eye, wit…

    Phase: PHASE1, PHASE2 • Sponsor: Ocugen • Aim: ⭐️ CURE ⭐️

    Last updated Aug 02, 2026 00:00 UTC

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