Marta Figueiredo, PhD, science writer —

Researchers have reported the case of two siblings with impaired CFTR function due to a deficiency in another chloride channel, called TMEM16A, that do not present symptoms associated with cystic fibrosis (CF). While there is evidence supporting either the activation or the suppression of TMEM16A activity as…

Problems with a protein called TAS2R38 are linked to a greater risk of severe sinonasal disease and Pseudomonas aeruginosa lung infections early in life in people with cystic fibrosis (CF), a study reported. These findings may help to identify high-risk patients, those with a dysfunctional form of TAS2R38, and improve approaches…

Most healthcare professionals are not fully prepared for cystic fibrosis (CF) end-of-life care, and further training and coordination between CF and palliative care teams would be helpful, according to a small, single-center survey in the U.K. Its researchers recommended a broader survey — one nationwide, or even international — to…

Iron deficiency is common among adults with stable cystic fibrosis (CF) and is significantly associated with anemia, vitamin A deficiency, antacids use, and moderate to severe lung disease, a study shows. Prevalence rates of iron deficiency were found to vary widely due to poor agreement between…

Streptococcus pseudopneumoniae should be included in the panel of opportunistic bacteria causing pulmonary exacerbations among those with cystic fibrosis (CF), according to a small French study. The study, “Streptococcus pseudopneumoniae, an opportunistic pathogen in patients with cystic fibrosis,” was published in the Journal of…

Enterprise Therapeutics‘ experimental compound called ETX001 was able to increase airway fluid secretion and mucus clearance in preclinical models of cystic fibrosis (CF), a study reports. ETX001 is a potentiator of a chloride channel called TMEM16A. Because TMEM16A’s production and function are independent of CFTR — the protein that…

Girls with cystic fibrosis-related diabetes (CFRD) are exposed to risk factors of early death — including chronic infections, poor lung function, and poor nutrition — at a significantly higher proportion and younger age than boys, a large European study shows. The findings highlight the importance of early diagnosis and effective treatment…

Eloxx Pharmaceuticals presented additional positive preclinical and clinical data for ELX-02, its lead treatment candidate for cystic fibrosis (CF), reinforcing the therapy’s safety and tolerability in humans and its ability to increase the levels of CFTR protein in preclinical models of the disease. Findings were presented in three posters and…

Researchers in Australia are growing mini gut and lung “avatars” derived from cells of children with cystic fibrosis (CF) to predict their response to current and potential medications. This platform may revolutionize the way clinicians treat CF patients, reducing the “trial-and-error” phase from the treatment process, and helping to…

Blocking the interaction between two molecules involved in immune restraining mechanisms — called PD-1 and PD-L1 — may be an effective therapeutic strategy to boost the immune system and treat chronic infections of Pseudomonas aeruginosa in cystic fibrosis (CF) patients, according to a recent study. The study, “…