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NCATS Director's Corner

Feb. 1, 2018

There are about 7,000 diseases officially defined as “rare,” or affecting fewer than 200,000 people in the U.S.; only a few hundred of these diseases have any approved treatment. Added together, these disorders are anything but rare: They affect 25 million people in the U.S., and approximately 50 percent of these patients are children. Most clinicians go through their entire careers without seeing a patient with one of these rare diseases; thus, patients and their families struggle first to get an accurate diagnosis, and then to get proper care and treatment, all while coping with frequently debilitating and costly illnesses.

Check out the rest of the NCATS Director’s Corner here: https://ncats.nih.gov/director.