Rare and Neglected Disease Research

Priorities Rare Disease Insert

We are currently aware of more than 7,000 rare diseases, which are defined as totaling fewer than 200,000 cases in the United States per year. While each rare disease has relatively few patients, cumulatively these diseases afflict 30 million Americans—roughly 10 percent of the population.

Because each disease affects so few people, drug companies have little financial incentive to research cures—as a result, effective treatments exist for fewer than three percent of all rare diseases.

At Notre Dame, we are committed to discovering treatments for these diseases because the value of a life cannot be measured by a simple return on investment. Whether a disease affects millions, thousands, hundreds, or even one person, our mission calls us to serve and to heal humanity.

In an effort to fill the huge hole created by the economics of healthcare and relieve the suffering of many people across the globe, the University of Notre Dame established two core research entities:

The Boler-Parseghian Center for Rare and Neglected Diseases

The Boler-Parseghian Center is an academic rare and neglected disease center that undertakes both basic and translational research. It works with families affected by rare diseases to combine studies of patient data and tissue with fundamental biological research in order to better understand the disease, identify molecular targets, and develop new diagnostics and treatments. The center has active pharmaceutical partnerships to accelerate the discovery and development of  drugs and therapies for rare and neglected diseases.

The Warren Family Center for Drug Discovery and Development

The Warren Center is a state-of-the-art resource for Notre Dame faculty that facilitates all stages of the discovery and development of potential therapeutics and establishes collaborations within the worldwide medical research community. A focal point has been the ongoing development of a library of compounds that may have therapeutic effects for other rare diseases.

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