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.

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ODC Newsletter 2019

ODC Newsletter 2019

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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.

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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