News

Children diagnosed with cystic fibrosis (CF) months after their birth have worse clinical outcomes than those diagnosed as newborns, a study reports. Its authors argue for improvements in CF diagnostic programs to detect these cases in infants as early as possible. The study, “Differences In Outcomes Between Early And Late Diagnosis Of Cystic Fibrosis…

Pharmaxis has entered into a research collaboration with the Woolcock Institute of Medical Research in Sydney, Australia, to develop an inhalation therapy that combines Pharmaxis’ Orbital Inhaler with a dry powder formulation of the antibiotic tobramycin. The resulting therapy is intended for patients with cystic fibrosis (CF).

Scientists at the University of Queensland, Australia, are focusing their efforts on what is one of world’s leading sources of antibiotic resistance — biofilm. Biofilms refer to the communities of bacteria and other microorganisms that adhere to surfaces in thin layers and secrete a protective coating that diminishes the effects of antibiotics. Biofilms are considered…

An infection caused by Mycobacterium abscessus, a species of multi-drug resistant mycobacteria, is a growing threat to cystic fibrosis (CF) patients, researchers in the U.K. warn. The infection shows signs of being increasingly contagious, and increasingly virulent. The study reporting on the bacterium — which can cause severe pneumonia and has been…

Enterprise Therapeutics recently announced that it has closed a £4 million (about $5 million) financing deal to accelerate the development of three new treatments  — all muco-regulatory therapies — for cystic fibrosis (CF), particularly, but also for chronic obstructive pulmonary disease (COPD) and severe asthma. “Respiratory disease remains an area of high unmet medical…

Celtaxsys recently announced that a second study describing the results of Phase 1 clinical trials evaluating acebilustat, an oral treatment for cystic fibrosis (CF), has been published. A Phase 2 study is underway and currently enrolling patients in the U.S., Canada and Europe. The research article is titled “Phase 1…

Children with cystic fibrosis (CF) who carry the F508del mutation in both CFTR gene copies showed improved lung function after treatment with Orkambi (ivacaftor/lumacaftor), according to results in a Phase 3 study developed by Vertex Pharmaceuticals. The study, in pediatric patients ages 6–11, evaluated the effect of Orkambi…

Scientists at Pennsylvania State University have developed a relatively inexpensive, easy-to-use system for diagnosing cystic fibrosis (CF), a technique that uses florescent light to measure levels of chloride in perspiration, which are known to be elevated in CF patients. The research was supported by the National Institute of Biomedical Imaging and…

ProQR Therapeutics is reporting that an early clinical study of  QR-010 in cystic fibrosis patients homozygous for the ΔF508 mutation in the CFTR gene met its primary endpoint. After four weeks of treatment, these patients showed a positive change in total chloride response, meaning that the CFTR protein was again working. In patients heterozygous for…

Verona Pharma recently announced that it has received its second Venture and Innovation Award from the U.K. Cystic Fibrosis Trust. The award will help fund a Phase 2 clinical trial to investigate RPL554 as a possible treatment of cystic fibrosis (CF). The trial, which expects to start recruiting CF patients during the first…