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Accelerating Cures to Rare Diseases

Relieving the financial burden for children participating in clinical trials for rare diseases

Duke University
Durham, NC Dr. Joanne Kurtzberg

In 2017 we established a Patient Advocacy Fund in the Pediatric Transplant and Cellular Therapy program (PTCT) program at Duke University to cover insurance copays and deductibles for children eligible to participate in potentially life-changing clinical trials when those costs are out of reach. Children from all over the U.S. and around the world travel to PTCT for access to clinical trials for rare life-threatening diseases. These are children with no other treatment options and often limited resources.