Joana Carvalho, PhD, managing science editor —

A panel of experts has released a series of recommendations for carers of children with chronic conditions, such as cystic fibrosis (CF), asthma, congenital heart disease, and diabetes, about how to manage these conditions from home. The new guidelines come at a much-needed time, as they may…

Vertex Pharmaceuticals announced that a reimbursement program covering Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftor combo) will be opening for eligible cystic fibrosis patients (CF) living in Switzerland. The program is part of a recent agreement reached between the company and the Swiss Federal Office of Public Health (FOPH) and…

Zikani Therapeutics has raised $7.5 million in Series A funding to advance work, using its TURBO-ZM technology platform, into potential therapies for rare disorders caused by nonsense mutations, including cystic fibrosis (CF). TURBO-ZM is a proprietary platform that enables Zikani to rapidly produce ribosome modulating agents…

Enrollment is continuing in a Phase 1/2 trial testing the safety and properties of AR-501, a potential inhalation treatment for chronic lung infections associated with cystic fibrosis (CF), Aridis Pharmaceuticals announced. The company also remains on track during the COVID-19 pandemic to report top-line trial findings: those in healthy volunteers…

Savara Pharmaceuticals has stopped patient enrollment into two of its ongoing clinical trials in cystic fibrosis (CF) patients — AVAIL and ENCORE — for safety reasons due to the COVID-19 pandemic. Steps will also be taken to ensure that those already enrolled in both studies continue…

Tiny particles, or nanoparticles, work better as transport and delivery vehicles for the RNA molecules central to some forms of gene therapy when plant-based relatives of cholesterol, called phytosterols, are included, scientists report. These findings are in the study, “Naturally-occurring cholesterol analogues in lipid nanoparticles…

Cystic Fibrosis News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other important events related to cystic fibrosis (CF) throughout 2019. As we look forward to bringing you more CF news this year, we present our 10 most-read stories of 2019. No. 10 –…

A triple combination of dirocaftor (PTI-808), Proteostasis Therapeutics’ investigational CFTR potentiator, with a proprietary CFTR amplifier and corrector led to significant improvements in lung function in patients with cystic fibrosis (CF) carrying the F508del mutation, according to topline data from a Phase 2…

The therapeutic triple combo elexacaftor (VX-445), tezacaftor, and ivacaftor leads to marked improvements in lung function and quality of life in people with cystic fibrosis (CF) carrying the F508del mutation, data from a Phase 3 trial show. The findings were reported in a study, “Efficacy and…

Researchers have discovered a stress response mechanism that allows Pseudomonas aeruginosa, a bacteria highly resistant to antibiotics that often causes lung infections in patients with cystic fibrosis (CF), to evade the action of antibiotics and to survive. The findings were reported in a study, “PQS Produced…