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Success Stories in Rare Disease Trials

Managing Rare Disease Clinical Studies in Ophthalmology: Highlighting first-hand clinical study success stories, this video excerpt focuses on the progress made in treatments for MacTel type 2, albinism, uveal coloboma, Stargardt Disease, and reticular pseudodrusen using innovative approaches like structural multi-modal imaging and biomarkers. From recruitment to regulatory strategies, the complexities of rare disease clinical studies and optimal solutions are addressed in this series of thought-provoking videos. The panel of experts from The National Eye Institute, University of Wisconsin and Emmes discuss everything from overcoming trial challenges to alternative study designs & endpoints.