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.
Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 clinical trial data for Trikafta® (elexacaftor/tezacaftor/ivacaftor) in children 6-11 years old with cystic fibrosis.
The Cystic Fibrosis Foundation has launched a new program that will fund 60 additional research coordinators to help speed the progress of CF clinical trials throughout its Therapeutics Development Network (TDN).
This morning, Vertex Pharmaceuticals announced the results from the Phase 3 clinical trials of ivacaftor (Kalydeco™) and lumacaftor (VX-809) in people with two copies of the F508del mutation.
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.
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.