While I’ve had the incredible opportunity to participate in clinical trials for cystic fibrosis, my nonsense mutations mean I can’t benefit from modulators. I was inspired by the North American Cystic Fibrosis Conference’s second plenary session, which discussed how we can evolve clinical trials to develop treatments for all people with CF.
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Mel Kotlyar
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5 min read
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.
Steph Hansen
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6 min read
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.
Schyler Kline
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5 min read
The additional funding will support a Phase 2 clinical trial for an inhaled antisense oligonucleotide drug for people with cystic fibrosis who have the splicing mutation 3849+10Kb C-to-T.
Press Release
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April 3, 2024
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3 min read