Every morning used to start the same way. My late daughter, Jasmine, would shuffle into the kitchen with wild-looking hair, rub the sleep from her eyes, and ask for applesauce with cinnamon. Before she could eat, though, she’d have to do her cystic fibrosis treatment regimen, which consisted of…
Respiratory symptoms are key drivers for starting Kaftrio (elexacaftor/tezacaftor/ivacaftor) among adults with cystic fibrosis (CF) who have stable, near-normal breathing ability, known as preserved lung function, a survey study in Europe has found. In surveying CF centers across the continent, the research team found that clinicians were more likely…
A therapy created using a miniaturized antibody can correct the functionality of the defective CFTR protein in people with cystic fibrosis (CF) caused by the F508del mutation. Preclinical data indicate that this nanobody can act in synergy with Trikafta (ivacaftor/tezacaftor/elexacaftor), an approved treatment for CF. In combination,…
Discussion
“Why do you keep talking about what you’ve been through?” Yes, I’ve actually been asked this before. No, I won’t stop talking about what I’ve lived through. It’s not for my own sake, but because people continue reaching out to me to say that sharing my story helps them feel…
Gene therapy using circular forms of DNA called plasmids may help treat lung disease in cystic fibrosis (CF), based on early lab studies, regardless of the disease-causing gene mutation, a study suggests. Optimized plasmids boosted production of the fully mature CFTR protein, which is missing or not working properly…
Discussion
During the summer of 2001, when my late daughter, Jasmine, was 9, she required a two-week hospital stay due to an acute pulmonary exacerbation — a complication of her cystic fibrosis (CF). Life was never the same after that. When I finally brought her home, the relief was…
Trikafta was associated with significant reductions in healthcare use, including hospitalizations related to cystic fibrosis (CF) complications affecting organs and systems outside the lungs, according to an insurance claims analysis. “Results of this study suggest a multisystemic benefit of [Trikafta] therapy with reductions in extrapulmonary…
Becoming educated and informed about cystic fibrosis is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your CF journey.
There is no cure for CF yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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