遗传和罕见疾病中心

**The National Center for Advancing Translational Sciences (NCATS)** is a unique and innovative research center within the National Institutes of Health (NIH) that was established to translate laboratory discoveries into treatments for patients. NCATS focuses on rare and neglected diseases, and its mission is to accelerate the discovery and development of new treatments for these diseases by bringing together researchers from a variety of disciplines to work together in a collaborative environment. **One of the highlights of NCATS is its Rare Diseases Clinical Research Network (RDCRN).** The RDCRN is a network of over 300 clinical sites across the country that are conducting research on rare diseases. The RDCRN provides a unique opportunity for researchers to collaborate on clinical trials and other research studies, and it has helped to accelerate the development of new treatments for rare diseases. **Another highlight of NCATS is its Therapeutics for Rare and Neglected Diseases (TRND) program.** The TRND program provides funding for research on the development of new treatments for rare diseases. The TRND program has helped to support the development of several new treatments for rare diseases, including treatments for cystic fibrosis, sickle cell disease, and Pompe disease.