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.

 
 
 
 

10 Crucial Years for Rare Disease Research

Penn Medicine News

January 1, 2022

A pioneer in the field of orphan diseases and gene therapy, James M. Wilson, MD, PhD, reflects on 10 years of leading Penn’s Orphan Disease Center.

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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.

Programs & Initiatives

 
 

Grant Opportunities

The ODC offers over 50 grant opportunities in 30+ disease areas annually to researchers across the globe, as well as within the Penn and CHOP community. Since 2011, our grant programs have funded $37.6 million in rare disease research.

JumpStart

The JumpStart program serves to establish and progress research agendas in emerging and neglected rare diseases. The JumpStart program partners with patient groups and families to address gaps by identifying key opinion leaders and introducing new researchers to a disease.

Million Dollar Bike Ride

The ODC is proud to partner with 30+ disease teams made up of patients, caregivers, foundations and family members who tirelessly raise much-needed funds for research grants.

 

Diseases We Study

 

Amyotrophic Lateral Sclerosis

CDKL5 Deficiency Disorder (CDD)

Mucopolysaccharidoses