The numbers are staggering. Approximately 7,000 rare diseases affect between 25 and 30 million, according to the National Institutes of Health. This equates to 1 in 10 people, or one on every elevator and four on every bus.
Yet for too many of them, treatment is out of reach because the government is standing in the way. That is why Right to Try 2.0 is a vital legislative reform. I’m a mother living in Arizona, and I’ve seen this need firsthand.
In August 2020, our family was forced to move to Milan, Italy, to access the one treatment option in the world that could save the life of our youngest daughter, who was diagnosed with a rare terminal illness that would otherwise take her life by age 6. This lifeline was based on a breakthrough technology that used genetics to customize a treatment for our daughter. But there was no way to get the treatment in the United States, at least not in time to save our daughter.
Kendra Riley is a business owner, mother of three, and rare disease advocate whose family has firsthand experience with cutting-edge, individualized medical treatments. She’s urging lawmakers around the nation to pass Goldwater’s Right to Try for Individualized Treatments reform. You can read more about the Riley family’s story at Kendra’s blog, The Riley’s Road to Italy.