Opinion: How the government can help lower the price — not just the cost — of cutting-edge gene therapies

At the bustling Chicago O’Hare airport, my luggage and I collided with an elderly couple. My heart sank when I realized my vision had failed me again. Because of a connective tissue disorder, my peripheral vision and balance were impaired, making such unintentional mishaps part of my journey.

Moments like these are stark reminders of the day-to-day issues that patients with rare diseases often face. I know this not only from my own experience living with a genetic disorder, but also from treating many patients and researching gene therapies for more than two decades.

Read the rest…