Almost two years ago, my nephew Gage learned he had Type 1 diabetes, a disease his older brother Hunter had been diagnosed with a few years before.  Shortly after the family got home from the hospital in Sioux Falls where Gage had learned to give himself shots and test his blood sugar levels, he was talking with his mom about what the diagnosis would mean.  At one point, he told her, “If God is going to heal me or Hunter, I hope he heals Hunter.  He’s had diabetes a lot longer than me.” 

What an incredible display of brotherly love!  Just a few months ago, another one of my nephews, Mitchell, was diagnosed with Type 1 diabetes as well.  It’s my hope and prayer that one day, because of the incredible research currently underway, we’ll be able to tell Hunter, Gage, Mitchell and young people like them that there is a cure; they can be healed.

On February 28, in observance of the 10th annual RARE Disease Day, people around the world turned their attention to diseases like this.  Throughout the day and the week that surrounded it, my team and I met with many South Dakotans whose families were forever changed by rare conditions. 

I spoke with one father, for instance, whose son had both Type 1 diabetes and Celiac disease, which our family has learned is a somewhat common combination as they are both autoimmune diseases (one of our nephews also has Celiac disease). The whole family has now gone gluten free to accommodate his son’s Celiac disease and with new technological advancements, he can monitor his son’s blood sugar levels almost minute-by-minute through an app on his phone.

We also met with two inspiring moms of children with cystic fibrosis and a father whose son has been living with a severe genetic disorder.  The disorder causes intellectual disability, behavioral and learning challenges, and various physical characteristics. Ongoing research offers more insight into how to manage life with the disease, but there is currently no cure.

While the syndromes and diseases take many different forms, I am consistently inspired by the resilience of those who live with these diagnoses and the caregivers who offer support.  It was with these families in mind that I helped pass the 21st Century Cures Act last year.  The bill was also approved by the Senate and eventually signed into law by the president.

The 21st Century Cures Act provided new support for research and biomedical innovation, specifically incentivizing the development of drugs and medical countermeasures for pediatric diseases.  Additionally, the legislation removed regulatory burdens that slow the pace of scientific advancement, modernized clinical trials, put patients at the heart of the regulatory review process, and streamlined processes that made it difficult to translate discoveries into FDA-approved treatments.

For more than a century, the United States has been on the leading edge of medical innovation and I’m committed to protecting this tradition for a century more.  With the 21st Century Cures Act now in place, I’m hopeful we’ve cleared a space where innovation can thrive and new forms of healing can take hold.

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