In the United States, orphan diseases represent a collection of disorders that afflict fewer than 200,000 individuals for any single disease type. There are more than 7,000 distinct orphan diseases.
In the aggregate, over 25 million people in the United States suffer substantial morbidity and mortality from orphan diseases. Despite this huge number, research in most disease types has lagged far behind other major areas due to a combination of technological and funding limitations.
The Orphan Disease Center of the University of Pennsylvania exists to facilitate and fund research, and develop transformative therapies for rare diseases with significant unmet need.
We aim to make rare disease research accessible, collaborative, and sustainable.