Most people with cystic fibrosis (CF) who carry mutations that make them ineligible for CFTR modulators are prescribed medications that improve mucus thinning and clearance. Antibiotic use is also high, and because most patients had pancreatic insufficiency, treatment with pancreatic enzyme replacement therapy is also common. That’s according…
News
Having COVID-19 had no clinically meaningful impact on lung function and nutritional status for people with cystic fibrosis in the year after an infection, a study across 33 countries indicates. According to the researchers, the “study is [a] global representation of the impact of COVID-19 on the health of…
Claire’s Place Foundation is gearing up to celebrate its 10th Annual Glow Ride for Cystic Fibrosis (CF) that’s set to happen Sept. 21 at Hermosa Beach, California. The milestone event aims to raise crucial funds to…
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…
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…
The U.S. Food and Drug Administration (FDA) has cleared a Phase 2 clinical trial testing ARCT-032, an inhaled therapy candidate, in people with cystic fibrosis (CF). Arcturus Therapeutics, the therapy’s developer, did not announce when the trial is expected to start. “The study is designed to evaluate the…
A report described episodes of skin rash associated with joint pain and inflammation, known as dermatitis-arthritis syndrome, in four females with cystic fibrosis (CF). The patients had similar symptoms, including pink, swollen, itching skin eruptions associated with joint pain. The researchers proposed cystic fibrosis dermatitis arthritis syndrome as a…
Gene therapy was found to work as well as a combination of the three CFTR modulators in Trikafta to restore the function to the CFTR protein, which is faulty in cystic fibrosis (CF), with the potential to work for all patients, regardless of their disease-causing mutation. That’s according…
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