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A New Era of Rare Disease Diagnostics & Therapeutics- Day 1
Penn’s Orphan Disease Center (ODC) is the first of its kind, a program dedicated to identifying promising therapies for oft-overlooked disorders and providing access to those therapies for patients of all populations. Led by world-renowned gene therapy pioneer Jim Wilson, the ODC has a track record of accelerating progress across a number of arenas, including infectious diseases, blood disorders, metabolic diseases, and neurologic disorders like Rett syndrome, CDKL5 deficiency disorder, and Duchenne muscular dystrophy. Join us as we take a look at where we have been – and where we are going.