Leber Congenital Amaurosis
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) [Phase 3]
The document describes an ongoing Phase 3 clinical trial evaluating Sepofarsen for Leber congenital amaurosis (LCA) type 10, which is curren…
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CLINICAL_TRIALS research · 2025-08-06Clinical Trial Update: Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis [Phase 1, Phase 2]
Ocugen's OCU400 is being studied in a Phase 1/2 trial to assess safety and efficacy in patients with Retinitis pigmentosa and Leber congenit…
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CLINICAL_TRIALS research · 2025-06-18Clinical Trial Update: Psychotherapy Group for Parents of Children With LCA [NA]
This clinical trial update notes a psychotherapy group for parents of children with Leber congenital amaurosis, and indicates the study is n…
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CLINICAL_TRIALS research · 2025-03-27Clinical Trial Update: Inherited Retinal Degenerative Disease Registry
A Foundation Fighting Blindness–sponsored inherited retinal degenerative disease registry is currently recruiting participants to collect in…
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CLINICAL_TRIALS research · 2024-09-19Clinical Trial Update: Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating HG004 for Leber congenital amaurosis due to RPE65 mutations is currently recruiting.
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CLINICAL_TRIALS research · 2024-09-19Clinical Trial Update: Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) [EARLY_Phase 1]
Early phase 1 clinical trial update (LIGHT) investigates a gene therapy for Leber congenital amaurosis, conducted by Xinhua Hospital and Sha…
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CLINICAL_TRIALS research · 2015-10-07Clinical Trial Update: Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/Phase 2 gene therapy protocol for retinal dystrophy due to RPE65 defects in Leber congenital …
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CLINICAL_TRIALS research · 2011-11-24Clinical Trial Update: Genetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal Dystrophy [NA]
A completed genetic study investigating patients with Leber congenital amaurosis and early severe retinal dystrophy.