Advocacy, Education, and Science to Improve Lives

Individually, rare diseases affect relatively few Americans. Collectively, they cause millions of people and their families to suffer. For many of these diseases, no treatment is available. For some, drugs or other therapies exist, but patients are unable to access them because the cost is too high.

KGI’s Center for Rare Disease Therapies addresses these issues. It raises awareness of rare diseases and promotes promising treatments. The center educates students to become biotech industry leaders who understand rare diseases and recognize the need for remedies. It brings together all of the parties who can make a difference and find solutions: patient advocacy groups, pharmaceutical and biotech companies, government agencies, research institutions, and nonprofit organization.

The center dedicates its advocacy, education and science to a singular purpose: making more treatments available to improve the lives of people with rare diseases.

The Center’s Mission

The mission of the Center for Rare Disease Therapies is to advocate new therapies for rare diseases and to increase awareness of rare diseases through education, science and ethical business practices. The center acts as a nonprofit catalyst that interacts with government agencies like the FDA’s Office of Orphan Product Development, patient advocates such as the National Organization for Rare Disorders and the Pharmaceutical Research and Manufacturers Association (PhRMA).

In the long term, the center aims to be a national and global resource for expertise, training and information related to rare diseases.