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A Multicenter, Prospective, Longitudinal, Observational Study In Children And Adults With Stargardt Disease Related Atrophy Secondary To Biallelic Mutations In The ABCA4 Gene
Rochester, Minn.
This multicenter, prospective, longitudinal, observational study in approximately 80 subjects with Stargardt disease secondary to biallelic mutations in the ABCA4 gene (STGD1) aims to evaluate prognostic factors of disease progression, and to further characterize the patient population for future clinical studies.
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A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease
Rochester, Minn.
The purpose of this study is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease.
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An Open-label, Multicenter, Two Part, Ascending Dose Followed By A Controlled Trial To Assess The Safety And Efficacy Of A Subretinal Administration Of AAVB-039 In Participants With Stargardt Disease (STGD1) (CELESTE)
Rochester, Minn.
The purpose of the 039-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-039 in participants with Stargardt disease secondary to a biallelic mutation of the ABCA4 gene. The study will also assess initial efficacy following AAVB-039 administration.
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Analysis of aqueous humor samples in participants with or without age-related macular degeneration
Rochester, Minn.
The purpose of this study is :to calibrate laboratory machinery to handle smaller volumes of ocular tissue samples (aqueous humor) and, ultimately, to compare the biomarkers present in such fluids between those with age-related macular degeneration (AMD) and those without AMD.
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Analysis of music therapy during ROP examination
Rochester, Minn.
The purpose of this study is to explore the use of live music therapy from Mayo Clinic's board-certified music therapists and recorded maternal singing to alleviate stress in infants undergoing ROP examinations.
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Analysis of the Mayo Clinic Intravitreal Injection Clinic
Rochester, Minn.
The purpose of this study is to collect data on the safety of not performing retinal imaging (e.g., optical coherence tomography, OCT) for patients receiving intravitreal injections. Analyze the outcomes, safety, and patient satisfaction of the Mayo Clinic Rochester Injection Clinic, where patients do not have retinal imaging or dilated eye examinations at every visit.
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Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants (Uni-Rare)
Rochester, Minn.
The purpose of this registry is to establish genetically and clinically well-characterized cohorts of patients across hundreds of genetic variants associated with retinal dystrophy (RD).
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