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Natural History Study in Retinitis Pigmentosa Caused by Mutations in the BEST1 Gene
The purpose of this study is to establish the natural history of of participants with BESTROPHIN 1 Vitelliform Macular Dystrophy. The blinding disorder Best V…
Best Vitelliform Macular DystrophyRetinitis Pigmentosa
Columbia UniversityNCT05809635
Phase 1
A Phase 1b/2a, Open-Label, Dose-Exploration Basket Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-BEST1 Administered in Patients With Either Autosomal-Dominant BEST1 Disease (Best Vitelliform Macular Dystrophy [BVMD]) or Autosomal-Recessive Bestrophinopathy (ARB)
The goal of this clinical trial is to learn if drug OPGx-BEST1 works to treat BVMD and ARB Bestrophinopathy. It will also learn about the safety of drug OPGx-B…
ARBBVMDAutosomal-Dominant Bestrophinopathy+1 more
Opus Genetics, IncNCT07185256