What is an Orphan Disease?
An orphan disease is a disease which affects a relatively small number of individuals and for which no drug therapy has been developed because the small market would make the research and the drug unprofitable.
Diseases are classified as orphan when they affect fewer than 200,000 people. However, as there are approximately 7,000 diseases now identified in this population, more than 25 million Americans are currently afflicted. Many of these diseases are caused by genetic mutations and are diagnosed in children. These diseases often appear before the age of two, and they can be debilitating and life-threatening, often times resulting in early death. An inadequate understanding of their underlying causes is one of the greatest obstacles standing in the way of new treatments and cures for orphan diseases.
Paving the Path to Cures
The Orphan Disease Pathway Project is a non-profit organization dedicated to finding cures and effective treatments for orphan, or rare diseases, and to facilitate research of such ailments. Providing financial assistance to organizations, hospitals, universities, research centers and doctors that are researching treatments for orphan diseases, is only part of what the Orphan Disease Pathway Project offers. The foundation also grants financial assistance to organizations that provide services to individuals and families who are affected by orphan diseases.
One of the largest endeavors of the Orphan Disease Pathway Project is its partnership with the Orphan Disease Center (ODC) at the University of Pennsylvania. The ODC was founded in 2011 by a generous donor whose vision matched that of Penn Medicine in developing treatments for these rare diseases. The ODC fills a crucial need by working closely with patient groups and foundations, pharma and biotech, and the academic community. The center offers a unique set of programs that seek to drive therapeutic development by identifying the most promising technologies, approaches, and experts, while simultaneously working to overcome barriers in the space. The ODC leads an international, coordinated effort to enable the research and development of safe, effective, and life-changing therapies for rare diseases across the globe.