Treatment with Trikafta (elexacaftor/tezacaftor/ivacaftor) cut sweat chloride levels in half among people with cystic fibrosis (CF), according to a real-world Danish study. Still, sweat chloride levels — a known biomarker of treatment responses in CF — were variable, and some treated patients still had levels out of the…
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The Rare Disease Cures Accelerator-Data and Analytics Platform — dubbed RDCA–DAP — has received new datasets that promise to accelerate research and the development of new therapies for cystic fibrosis (CF). The data was generated by the Cystic Fibrosis Therapeutics Development Network (TDN), known for its clinical trials…
An infant boy, who was first seen with recurrent infections and a failure to thrive, was diagnosed with cystic fibrosis (CF) caused by a rare genetic mutation, a case study reports. “This case underscores the importance of considering CF even when classic symptoms are absent, as genetic mutation can…
A 3-year-old boy who carries a new combination of two mutations in the CFTR gene, but with no clinical signs of cystic fibrosis (CF), was described by researchers in a case report. “The case presented has no clinical symptoms so far and doesn’t meet the criteria for a…
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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