My son, Arjun, has had the deck stacked against him in some ways. He was born early, with cystic fibrosis, and needed surgery. His resilience, and my hopes for his future, keep me inspired.
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My son has had a problem with eating ever since he was born prematurely and diagnosed with cystic fibrosis. After a lot of stress and anxiety about reaching his weight goals, we finally enlisted an occupational therapist, who helped him learn to love eating.
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.
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.
Legislation Would Remove Financial Penalties for Participating in Research Studies
New Bill Would Remove Financial Penalties for Participating in Research Studies
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.
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.