Rare diseases impact 1 in 10 Americans, and over 400 million people worldwide. The current development and FDA approval process can cost hundreds of millions of dollars – and take an average of 15 years – to develop one therapy. For the 30% of children diagnosed with a rare disease, who won’t live to see their 5th birthday, this wait is unthinkable. Meanwhile, prolonged – and unnecessary – diagnostic delays and inadequate treatment protocols yield increased healthcare costs a