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

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 will review 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.   

Learn more and register!



Million Dollar Bike Ride 2020 Pilot Grants are now available!

Submit your LOI today!

The Orphan Disease Center is offering 38 research opportunities focusing on 29 different rare diseases. This program provides a one-year grant to support research related to a rare disease represented in the 2020 Million Dollar Bike Ride. Number of awards and dollar amounts vary per disease based on fundraising totals by each disease team. 



LOI’s are due Friday, September 18, 2020 by 8PM EST

This Request for Applications is open to the international research community

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

Jump Start

Million Dollar Bike Ride

International Patient Registry

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