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A Multicenter, Prospective, Longitudinal, Observational Study in Children and Adults with Stargardt Disease Secondary to Mutation in the ABCA4 Gene

Primary Objectives• To evaluate prognostic factors of disease progression in subjects with Stargardt disease secondary to mutation in the ABCA4 gene • To further characterize the STGD1 patient population for future interventional clinical studies

Universal Rare Gene Study: A Registry and Natural History
Study of Retinal Dystrophies Associated with Rare Disease-Causing Genetic Variants

IRDs are genetically diverse (280 causative genes have been identified to date) (https://sph.uth.edu/retnet/) and have vastly different clinical manifestations, including age of onset, severity of disease, rate of progression, and structural and…