Penn raises $10 million for 'orphan' disease effort

Donations totaling $10 million, mostly from a single anonymous donor, will allow the University of Pennsylvania's Perelman School of Medicine to create an interdisciplinary center focused on finding and disseminating treatments for orphan diseases, the school announced Tuesday.

Each "orphan" disease affects a relatively small number of people - fewer than 200,000 Americans - and that makes them less attractive targets for big pharmaceutical companies than more common maladies offering a bigger potential market.

Penn said there are 7,000 orphan diseases affecting 25 million Americans. Many are caused by genetic mutations and are diagnosed in children.

The Penn Center for Orphan Disease Research and Therapy will lead an international, coordinated effort to attack these diseases. Glen Gaulton, chief scientific officer at the Perelman School, said the new center would help small organizations devoted to a single disease design grants and share scientific information needed to conduct research.

It will also offer a new robotic drug-screening laboratory that will allow researchers to rapidly look for possible treatments among existing compound libraries, including those at pharmaceutical companies.

Gaulton said drug companies are interested in learning whether orphan diseases respond to treatments the companies have already developed.

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