NCATS CHALLENGE AWARD-NCATS RARE DISEASES ARE NOT RARE

Investigators

Abstract

If you know 10 people, chances are you know someone with a rare disease. There are about 7,000 different rare diseases that affect an estimated 30 million people in the United States. Some difficulties with rare diseases are that they are hard to recognize, are often hidden conditions, and most do not currently have ongoing medical research. This Challenge seeks to bring attention to rare diseases so that they can gain more medical research interest, thereby improving the lives of people with rare diseases. Science, especially genetic medicine, has moved forward to the point now where treatments are possible-such as gene therapies, 3-D printing (devices, tissues, and organs), and new drugs. Everyone deserves a chance at an effective medical treatment-whether for a common disease or a rare one-so let's get the word out!