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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.

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