Marta Figueiredo, PhD, science writer —

A complete analysis of the CFTR gene, which is defective in cystic fibrosis (CF), identifies potential disease-causing mutations that would be missed by standard testing, as well as variations that may explain differences in disease course and treatment responses, a study of more than 5,000 CF patients shows. Still,…

Eloxx Pharmaceuticals’ experimental therapy ELX-02 was generally safe and resulted in a significant reduction in sweat chloride levels — a biomarker of cystic fibrosis (CF) — in patients carrying the most common CF-causing nonsense mutation, according to top-line data from a Phase 2 clinical program. While the short…

Aceragen reached an agreement to acquire Arrevus, including its late-stage clinical program of ARV-1801 — now called ACG-721 — an experimental oral therapy for pulmonary exacerbations in cystic fibrosis (CF) patients. Aceragen is planning to launch a Phase 2/3 clinical trial in the second half of 2022…

A team of researchers from the Michigan State University (MSU) College of Human Medicine and Spectrum Health will use a $2.1 million grant from the National Institutes of Health (NIH) to advance preclinical research on a gene therapy for cystic fibrosis (CF). The four-year award was given to the…

Cystic fibrosis (CF) patients infected with the virus that causes COVID-19 are at a higher risk of hospitalization, a need for intensive care, and kidney injury relative to COVID-19 patients without this disease, according to a large, multi-center study in the U.S. While previous research reported that CF…

The implementation of newborn screening for cystic fibrosis (CF) in the U.S. allowed for early diagnosis and clinical evaluation of affected infants, and reduced rates of serious lung infections and hospitalizations over time, according to data from the first nine years of universal screening. However, early nutritional deficits…

The COVID‐19 pandemic increased socioeconomic instability among people with cystic fibrosis (CF), mostly in terms of employment and ability to afford food, according to data from a single-center U.S. study. Notably, patients who were employed prior to the pandemic appeared to be the most affected, reporting job loss and concerns…

Differences in Psl, a complex sugar molecule produced by Pseudomonas aeruginosa, contribute to the bacterium’s resistance against the antibiotic tobramycin in children with cystic fibrosis (CF), a study shows. While tobramycin-resistant P. aeruginosa produced similar levels of Psl to those that were effectively eradicated with the antibiotic, its Psl molecules…

Researchers at the University of Massachusetts (UMass) Amherst have developed a new method of RNA production that leads to higher yields and purity of RNA molecules, likely making it more cost-effective than current approaches. Their work may be of importance to the production of RNA-based therapies for cystic fibrosis (CF)…

Assessment, documentation, and education regarding cannabis and cannabidiol (CBD) use among cystic fibrosis (CF) patients varies across centers in the U.S., according to a survey of nearly 300 CF healthcare providers. This variability may be related partially to the different laws between U.S. states regarding these controversial substances, the…