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Penn medical school to create orphan drug center with $10M gift

The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania said Tuesday it has received a $10 million gift from an anonymous donor that will be used to create an interdisciplinary center focused on discovering novel treatments for rare, so-called “orphan” diseases.

Orphan diseases are those that affect fewer than 200,000 people. An estimated 7,000 orphan disease afflict more than 25 million people in the United States.

Penn said the funding recognized the institute’s success during the first five years of… more

Penn officials said 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.”

The center’s resources will include a robotically controlled drug-screening laboratory that Penn said will enable researchers worldwide to quickly probe existing compound libraries for effective treatments.

“The research and development marketplace is not designed to optimally support research to develop the therapies so desperately needed for orphan diseases,” said Glen N. Gaulton, executive vice dean and chief scientific officer at the Penn medical school. “Penn’s new Center for Orphan Disease Research and Therapy will build not only strong collaborative relationships throughout Penn, but also with other leading academic medical centers, as well as public and private institutions — all designed to translate innovative research into the clinic. There’s simply nothing else like it.”

Arthur H. Rubenstein, who last month stepped down as dean of the Penn medical school, will serve as acting director of the center while an international search for a permanent director is conducted.

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