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Clinical Trials for Retinal Dystrophies

• SAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects
• Disulfiram for Treatment of Retinal Degeneration
• An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
• BEOVU in the Treatment of Dystrophy-related Macular Neovascular Degeneration
• Oral Metformin for Treatment of ABCA4 Retinopathy
• A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene
• Minocycline Treatment in Retinitis Pigmentosa
• Intravitreal Ranibizumab (Lucentis®) in the Treatment of Non-leaking Macular Cysts in Retinal Dystrophy
• Phase 1 Safety Study of ALK-001 in Healthy Volunteers
• Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65