The mother of a young boy with Progeria has paved the way to the world's first clinical trial into a potential treatment of the disease, which will require recruiting more than half of the 42 known patients in the world. The case underscores both the promise of new genetic research into potential treatments as well as the challenges researchers face in studying rare diseases. Progeria affects only one in every four million to eight million children, most of whom die from a stroke or heart attack by the age of 13.
- here's the article from The Wall Street Journal (sub. req.)