News

Treatment for 14 days with Proteostasis Therapeutics’ PTI-801 in cystic fibrosis (CF) patients also being treated with Orkambi (lumacaftor/ivacaftor) led to statistically significant improvements in sweat chloride, body mass index, and rescued blood glucose levels in a subgroup of patients with diabetes, Phase 1 trial data show. PTI-801 is being…

Real-world results from the largest analyses of Vertex Pharmaceutical’s cystic fibrosis’ treatment Kalydeco (ivacaftor) done in CF patients to date confirm the therapy’s ability to significantly lower the risks of mortality, transplants, hospitalization, and pulmonary exacerbations compared to untreated patients. These results were presented today at the 41st European Cystic…

Results of the Phase 3 ARRIVAL study show that Kalydeco (ivacaftor) has the potential to modify the course of toddlers with cystic fibrosis. The trial’s most recent finding were discussed at the 41st European Cystic Fibrosis Society (ECFS) Conference taking place in Belgrade, Serbia, through June 9.

Exposure to tobacco smoke is a key contributor to worsening lung function in young children with cystic fibrosis (CF), researchers report in a study that highlights both the importance of routine checks of lung function in children, and smoking cessation programs as a part of CF care. The study’s results…

Preliminary clinical trial data of three investigational next-generation CFTR correctors, being developed by Vertex Pharmaceuticals, provides the first demonstration of their potential to improve the outcome of cystic fibrosis (CF) patients. Trial findings were presented today at the 41st European Cystic Fibrosis Society (ECFS) Conference, taking place…

One-year survival after a lung transplant of cystic fibrosis (CF) patients chronically infected with antibiotic-resistant bacteria is similar to those without infections, according to a retrospective analysis of data from the International Society of Heart and Lung Transplantation (ISHLT) Thoracic Transplant Registry. Results of the study were shared today…

Results of a Phase 2 trial showed that treatment with Spyryx’s investigative drug SPX-101 can improve lung function in patients with cystic fibrosis, regardless of their background genetic mutation causing the disease. The most recent trial data will be discussed at the 41st European Cystic Fibrosis Society…

Clinical centers around the world differ in the standards they use when considering cystic fibrosis (CF) patients with nontuberculous mycobacteria (NTM) infections for lung transplants, and few have a clear written policy, a U.K. study shows. These findings reflect the need for standardized guidelines for listing cystic fibrosis…

Proteostasis Therapeutics will present an update on its cystic fibrosis (CF) clinical programs — which include an amplifier, a corrector, and a potentiator of the CFTR gene — at the upcoming 41st European Cystic Fibrosis Society (ECFS) Conference, June 6-9 in Belgrade, Serbia. In two presentations, the company will cover the most recent advances…

Two cystic fibrosis (CF) patients being treated for lung infections related to Mycobacterium abscessus — a multidrug-resistant nontuberculous mycobacteria (NTM) — using inhaled granulocyte-macrophage colony stimulating factor (GM-CSF) now both show signs of cure, Savara Pharmaceuticals announced. M. abscessus accounts for about 35 percent of NTM infections in cystic fibrosis patients in North America, researchers…