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.
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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.
There are two main types of clinical studies in CF research. Both are important to move research forward and provide the best outcomes for people with CF.
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.
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.
Have questions about clinical trials? The Clinical Trial Navigator is a person who can help you get the answers you need.
Clinical trials are critical to developing new treatments for CF. So, how exactly does an experimental drug become an approved therapy?
There have never been more opportunities to help develop new drugs for cystic fibrosis than there are today. When you are deciding whether to participate in a