National Center for Advancing Translational Sciences - Rare Diseases

The National Center for Advancing Translational Sciences' (NCATS') mission is to turn research observations into health solutions through translational science. We work to develop or enhance the development, testing, and implementation of diagnostics and therapeutics for a wide range of diseases and conditions. Key approaches include understanding what’s similar across diseases to spur multiple treatments at a time, developing models that better predict a person’s reaction to treatment, enhancing clinical trials so results more accurately reflect the patient population, and leveraging real-world data and data science approaches to address public health needs. Facilitating these approaches are our robust partnerships with other government agencies, including other NIH institutes, centers, and offices; industry; academia; nonprofit organizations; and patients, patient advocates, and other communities. NCATS' vision is to bring more treatments for all people more quickly.