Marta Figueiredo, PhD, science writer —

The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) as the first-ever therapy to treat the underlying cause of cystic fibrosis (CF) in 1-year-old toddlers, Vertex Pharmaceuticals announced. “Parents and physicians now have a medicine to treat the underlying cause of CF in patients as young as one…

A therapy whose developers say could revolutionize the battle against antibiotic-resistant lung infections in cystic fibrosis patients eliminated resistant Staphylococcus aureus in a lab, a study reports. The finding dealt with Synspira’s polycationic glycopolymer SNSP113 as a stand-alone treatment. The study also showed that the therapy increased the punch of antibiotics used to counter…

One of the most frequently mutated genes in cancer, PTEN, in involved in the repeated lung infections that trouble cystic fibrosis patients — not because PTEN is abnormal in CF, but because it is unable to work with CFTR gene, which is mutated in this disease, to help clear the…

Sevion Therapeutics and Eloxx Pharmaceuticals announced that a first healthy subject has been dosed in a Phase 1b clinical trial assessing the safety, tolerability and drug properties of ELX-02 as a potential treatment of several genetic diseases caused by nonsense mutations, including cystic fibrosis (CF). Nonsense mutations are…

Cystic fibrosis (CF) patients have more bacteria but lower bacterial diversity in their lungs than people without CF — yet bacterial diversity diminishs with age in CF patients, a large multicenter study found. The study, “Airway microbiota across age and disease spectrum in cystic fibrosis,” appeared in…

Scientists at the University of North Carolina (UNC) School of Medicine developed a model of cystic fibrosis (CF) that may help to test current and future CF therapies. This model, showing how airway cells respond to environmental factors, also brought new insights into the thick mucus observed in CF patients. The…

Italian researchers have discovered a molecule that prevents cystic fibrosis patients’ anti-inflammatory and anti-bacterial defenses from working as well as they should. Scientists may be able to target the molecule, microRNA-181, to develop CF therapies. The team published their study in the journal Nature. The title is  “MicroRNA-181b…

The European Union’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for Orkambi (ivacaftor/lumacaftor) as a therapy for cystic fibrosis (CF) in children. The ruling is specifically for pediatric patients aged 6 to 11 with two copies of the F508del mutation in the…