Assuring access to transformative therapies for all rare disease communities.

Connecting the dots

Research and funding for orphan diseases is grossly unmet. ODC provides technological and educational resources, identifies funding opportunities, and fosters therapeutic development and innovative research.


September Webinar Series

Thank you all who participated!

Spread over four weekly sessions in September 2020, co-hosted by the Orphan Disease Center at the University of Pennsylvania and Amicus Therapeutics.  The series reviewed the role of patient registries and high quality natural history data in advancing clinical development of rare disease therapeutics, with a focus on potentially curative technologies such as gene therapy and genome editing.  

For more information and session resources click here

To view recorded sessions click here




Orphan Disease Center 2020 Fundraiser (Short Event Version)

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.

Programs & Initiatives

Grant Opportunities


Million Dollar Bike Ride

In The News

February 26, 2019

Amicus Establishes Global Research and Gene Therapy Center of Excellence in Philadelphia

CRANBURY, N.J. and PHILADELPHIA, Feb. 26, 2019 (GLOBE NEWSWIRE) -- Amicus Therapeutics today announced it is establishing a new Global Research and...

October 25, 2017

Meaningful Data Drives Better Trial Design and Faster Drug Approvals

But data for rare diseases can be difficult to obtain, which is why patient registries are playing an increasingly important role in helping bridge...

Meaningful Data Drives Better Trial Design and Faster Drug Approvals