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Center for Orphan Research and Therapy at Penn Medicine

The Center for Orphan Disease Research and Therapy at Penn Medicine would like to invite all researchers at Drexel University whose research includes Rare/Orphan Diseases to join the center.  The newly created Center was launched 8 months ago with goal of finding cures and making life better for all who suffer with Rare Orphan Diseases.

Orphan diseases represent a collection of disorders that afflict less than 200,000 individuals for any single disease type, yet 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.  Recognizing these challenges, the Penn Center for Orphan Disease Research and Therapy (CODRT) was formed with the mission of facilitating and expediting the development of novel therapies for orphan diseases.  The Center will achieve this mission through promoting development of novel therapeutic strategies and translating these into the clinic, building on partnerships among investigators, academic institutions, industry, and federal and private funding agencies.

Despite being a newly established Center at the University of Pennsylvania, members include participants who share the same vision from Australia, Canada, China, Italy, the United Kingdom, and many universities throughout the United States.  We encourage all faculty whose research incudes Rare Diseases to join by filling out the survey on the membership web page. There is no cost to join.  See below for more information on becoming a member, research funding opportunities, upcoming center events, and related web links.


The Penn Center for Orphan Disease Research and Therapy (CODRT) invites faculty members at Drexel University who conduct research in rare diseases to join the Center. The CODRT will provide resources and funding opportunities to Center members with demonstrated expertise in rare disease research and/or therapy development. Trainees interested in rare disease research can join as Associate members.

Center membership will provide:

  • Connections between researchers and clinicians at Penn/CHOP and other intuitions who are working on orphan diseases
  • Access to drug repurposing screens as well as small molecule screens
  • Sources of funding for research on orphan diseases
  • Access to small and large pharmaceutical companies to partner in the development of new therapeutics for orphan diseases
  • Access to animal models of orphan diseases

For membership information:

Research and Funding:

The Center also is highlighting a recent Request for Applications to support research on the development of improved therapies for patients with syndromes dues to MPS I including Hurler, Hurler-Scheie and Scheie. 

For Research information:

Upcoming Center Events:

Please join us Thursday, November 21, 2013 for the First Annual Center Retreat, “Charting Paths Forward in Rare Diseases”. The day will start with an overview of the center followed by presentations on Center partners and opportunities in rare disease research, drug development, and biomarker discovery.


Guest speakers will include: John F. Crowley, JD, MBA, Chairman and CEO, Amicus Therapeutics; Larry Gold, Ph.D., Chairman, Founder, and CEO of SomaLogic, Inc.; Stephen C. Groft, Pharm.D, Director, Office of Rare Diseases (ORD) at the National Institutes of Health; John C. McKew, Ph.D., Chief, Therapeutics Development Branch, NIH Center for Translational Therapeutics; Bill Leinweber, President and CEO, National Disease Research Interchange; Dave C Schultz, Ph.D., Facility Director, Molecular Screening Facility, The Wistar Institute.


For information and registration:   

In the Spring of 2014, the Center will sponsor two events to focus awareness on orphan diseases and research.  On Friday, May 2, 2014 we will hold the First Annual Symposium on Orphan Diseases followed by a Cycling Event for Rare Diseases on Saturday May 3.  The cycling event is designed to raise money and awareness for international rare disease research. The event will begin and end on the Penn campus with optional 10, 30, and 70 mile routes. Even if you are not a cyclist, please join us for the day’s activities.

For further information on the Penn Center for Orphan Disease Research and Therapy, please visit us at