Finding Treatments for Children With Rare Diseases

Finding the cause of a rare disease is often like looking for a needle in a haystack. More than 80 percent of rare diseases are genetic in origin^[i], and dedicated medical researchers can spend their careers looking for just one tiny defect in one gene that could explain hugely complex conditions.

The World Health Organization tells us that between one and two percent of children are born with this kind of rare disease, but more than 90 percent of…