Post
The Cost of Drugs for Rare Diseases Is Threatening the U.S. Health Care System
Harvard Business Review
No parents should have to watch their child die, yet my former colleague “Will” and his wife “Mary” watched powerless as two of their children succumbed to spinal muscular atrophy (SMA). “Isaac” and “Lizzy” were never able to sit, talk, or eat on their own, and each passed away as toddlers.