The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania announces the launching of a first-of-its-kind interdisciplinary center focused on discovering novel treatments for orphan diseases. The Center will bring together, without institutional walls, all necessary approaches to attacking and treating orphan diseases: establishing dedicated research support facilities, translating findings into therapies, fostering targeted grant awards, and educating physicians and researchers.

Formation of the new Penn Center for Orphan Disease Research and Therapy was catalyzed by a $10 million gift from an anonymous donor, whose vision matched that of Penn Medicine in developing treatments for these diseases. The Center will fill a crucial need by providing the core laboratories, techniques, collaborative relationships, and expertise to lead an international, coordinated effort in the eradication of orphan diseases. Key among these resources is a state-of-the-art, robotically controlled drug screening laboratory that enables researchers from around the world to rapidly probe existing compound libraries for effective, orphan disease treatments.

Diseases are classified as orphan when they affect fewer than 200,000 people. However, as there are approximately 7,000 diseases now identified in this population, more than 25 million Americans are currently afflicted. Many of these diseases are caused by genetic mutations and are diagnosed in children. Research in many orphan diseases has lagged behind other major disease categories, such as diabetes and cardiovascular disease, in part because of a relative lack of technical expertise and funding mechanisms. Penn's Center will specifically address these needs.

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