In lab studies, researchers have identified rare mutations in the CFTR gene — which are the cause of cystic fibrosis (CF) — that are not currently eligible for CFTR modulator therapies but might be treatment responsive. The researchers hope the findings will provide guidance to developer Vertex Pharmaceuticals…
News
The U.S. Food and Drug Administration (FDA) has given the go-ahead for a Phase 2 trial testing the inhalation therapy SPL84 in people with cystic fibrosis (CF) caused by a specific mutation called 3849+10 kb C-to-T. Therapy developer Splisense welcomed the FDA’s clearance of the clinical trial, and…
For adults with cystic fibrosis (CF), the symptoms with the biggest impact on quality of life include pain, fatigue, shortness of breath, and mental health challenges, leading researchers to recommend routine screening to assist CF patients who could benefit from support. “Screening for prevalent symptoms that affect quality of life…
Bacteria in the airways of people with cystic fibrosis (CF) can interfere with the growth of Pseudomonas aeruginosa by secreting acetic acid, a main ingredient in vinegar. That’s according to “Airway commensal bacteria in cystic fibrosis inhibit the growth of P. aeruginosa via a released metabolite,”…
4D Molecular Therapeutics (4DMT) is planning a Phase 3 clinical trial that, if results are positive, will be the basis of applications seeking approval of the company’s inhaled gene therapy 4D-710 as a treatment for people with cystic fibrosis (CF) who can’t take CFTR modulators. 4DMT…
A grant of $500,000 in Australian dollars (around $326,000) will support researchers there in the development of antibiotics formulated with liquid crystalline nanoparticles (LCNPs), the better to eliminate hard-to-treat lung infections in people with cystic fibrosis (CF). “By overcoming the processes that cause drug resistance and uncontrollable infection, this…
Abnormal glucose tolerance is common in young cystic fibrosis (CF) patients. Early screening, starting at the age of 5, may lead to better health outcomes by making it possible to intervene early, a meta-analysis study found. “Abnormal glucose tolerance affects every third child” and “every second adult” with CF,…
Elevated levels of the pro-inflammatory signaling protein galectin-3 in the bloodstream of children with clinically stable cystic fibrosis (CF) significantly correlated with worse lung function, according to a study. The findings suggest galectin-3 may serve as a noninvasive biomarker to predict lung disease outcomes in children with CF, the…
A history of pancreatic inflammation, diabetes, high blood pressure, and high cholesterol may increase the risk of pancreatic cancer in people with cystic fibrosis (CF), according to a large database report. These patients also had a higher prevalence of known risk factors for pancreatic cancer, including smoking, alcohol consumption,…
People with advanced liver disease associated with cystic fibrosis (CF) have more pro-inflammatory bacteria in their feces relative to those without CF- associated liver disease, a study shows. The findings suggest similar changes in the gut microbiota, or the populations of friendly microbes that naturally live in the intestines. This…
Your CF Community
Visit the Cystic Fibrosis News Today forums to connect with others in the CF community.