Steve Bryson, PhD,  science writer—

Vertex Pharmaceuticals has reached reimbursement agreements with health authorities in France and Italy for its various cystic fibrosis (CF) treatments, including Kaftrio (ivacaftor/tezacaftor/elexacaftor), a next-generation therapy approved for use with ivacaftor. With these agreements — which bring the treatments into each country’s public health program — about 3,000 CF patients…

Reduced levels of the VIP hormone in the pancreas due to low nerve supply may contribute to the development of early diabetes in people with cystic fibrosis (CF), a study in mice suggests. The study, “VIP reduction in the pancreas of F508del homozygous CF mice and early signs…

Two months of treatment with Kalydeco (ivacaftor) by cystic fibrosis (CF) patients who carry a CFTR gating mutation significantly increased the diversity of bacteria in the gut but not in the respiratory system, a small study reported. A…

Human mesenchymal stem cells (hMSCs) effectively treated infections caused by non-tuberculous mycobacteria in cell-based tests and a mouse model of cystic fibrosis (CF), a study revealed.  Although this strategy may help treat those infections in people with CF and other lung…

Antibiotic-resistant infection with Mycobacterium abscessus bacteria in people with cystic fibrosis (CF) can evolve rapidly to become more virulent, a genetic analysis suggests.  The findings support early testing and treatment of lung infections before symptoms emerge to…

Eloxx Pharmaceuticals has announced that it’s now added the approved cystic fibrosis (CF) therapy Kalydeco (ivacaftor) to its global Phase 2 clinical program for ELX-02. Ongoing ELX-02 Phase 2 clinical trials, launched in 2019 with four treatment…

A soft skin sticker that can rapidly measure chloride levels in sweat, which are elevated in cystic fibrosis (CF) patients, may help avoid delays in diagnosis and treatment associated with current testing methods, especially in young children, a study reported.  The so-called sweat…

Eloxx Pharmaceuticals has acquired Zikani Therapeutics and will expand its research program in ribosomal RNA-targeted therapies, with the goal of treating diseases such as cystic fibrosis (CF) caused by a specific type of genetic mutation. …

A precise gene-editing tool that reverses single DNA mutations may lead to genetic therapies for people with cystic fibrosis (CF), a study suggests. The study, “Programmable C:G to G:C genome editing with CRISPR-Cas9-directed base excision repair…

Molecular analysis of single cells lining the digestive tract found high CFTR activity — the gene associated with cystic fibrosis (CF) — in some cells in the duodenum, or first part, of the small intestine. A comparison of cells lining the stomach of…