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.

On behalf of the Orphan Disease Center and Gene Therapy Program at the University of Pennsylvania, we are enormously proud of our fearless leader, Dr. Jim Wilson, for his nomination of the very prestigious Sanford Lorraine Cross award. What an extraordinary achievement! 

Dr. Jim Wilson: Sanford Lorraine Cross Award Nominee

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

December 06, 2017

FAST Summit & Gala Lures 1,300 to Chicago, Raises $4 Million for Angelman Syndrome Research

This year’s FAST Summit & Gala in Chicago generated a record $4.1 million for Angelman syndrome research — more than triple the $1.3 million raised at...

FAST Summit & Gala Lures 1,300 to Chicago, Raises $4 Million for Angelman Syndrome Research

November 29, 2017

CDKL5 Forum

CDKL5 stands for cyclin-dependent kinase-like 5, a protein whose gene is located on the X chromosome. The CDKL5 gene provides instructions for making...

CDKL5 Forum

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