Participating in the RARE study gave me an opportunity to play a role in finding a therapy that one day could help me and other people with CF who have two nonsense mutations.
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Participating in clinical trials can be scary, but the sense of empowerment you get from knowing that you are contributing to a cure outweighs any second thoughts.
Last year -- just one week before my 12th birthday -- my doctor reached out to my family and asked me if I wanted to enroll in a clinical trial. Here's why I decided to say yes.
From a young age, I was determined that cystic fibrosis would never stop me from pursuing my dreams in the medical field. It wasn’t until I hit a road bump in pharmacy school that I realized my CF could actually give me a unique advantage.
Back in 2015, my genetic sequencing revealed one nonsense mutation. But recently, I learned I have another nonsense mutation that may not have been identified when my genes were first sequenced. As more mutation-dependent therapies are developed, it’s crucial that people with CF have accurate information so they can make informed decisions.
For those of us who can’t take modulators, it can sometimes feel like you’ve been left behind. But I have found hope and motivation by participating in clinical trials.
As cystic fibrosis damaged my lungs, I did all I could to make it to my university graduation before I underwent lung transplant.