The COVID-19 pandemic has brought the question of life and death to ordinary people who have rarely had to think about it before. The ability of biotechnology to provide two potent vaccines also reminds us of how much our new technology can change the way we view disease and the power of a new treatment. For rare disease patients before any pandemic, life and death or disability was a daily concern, and only the latest advances in science are now providing new options that can change their lives. The great advances in treatment technology have now opened the door to first-ever treatments for hundreds of rare diseases that affect up to 10% of the U.S. population. We are living in the golden age of treating rare disease patients.
