GNS Healthcare Blog
The Orphan Drug Act of 1983 defined a rare disease as one that affects less than 200,000 people. According to the NIH National Center for Advancing Translational Sciences, there are now 7,000 such diseases afflicting between 25-30 million people in the U. S. and an estimated 350 million worldwide.
Finding therapies for rare diseases not only helps those affected, but according to studies from the NIH Undiagnosed Diseases Program, new discoveries made from studying these diseases can also provide insights into more common conditions.
At Alexion, we are interested in understanding the rare...