News

Europe’s largest annual gathering of cystic fibrosis (CF) experts ended June 9, capping four days of speeches, workshops, and poster presentations that attracted 1,832 participants from 56 countries. The largest contingents at the 41st European Cystic Fibrosis Conference, held in Belgrade, Serbia, came from the United Kingdom (304) and Serbia itself…

Preclinical studies show that Eloxx Pharmaceuticals’ lead therapy candidate ELX-02 can reverse the effects of CFTR gene nonsense mutations in mice and cystic fibrosis (CF) patient-derived organoids. These findings further support the clinical development of ELX-02 as a treatment for CF patients with CFTR nonsense mutations, which represent…

The European Cystic Fibrosis Society presented three scientists — one Portuguese, one Mexican, and one German — with the organization’s 2018 “Young Investigators Awards” on June 9, the last day of its 41st European Cystic Fibrosis Conference (ECFS) in Belgrade, Serbia. The winners are Iris Silva, Samuel Lara Reyna, and…

AmpliPhi Biosciences’ investigative therapy AB-PA01, in combination with antibiotics, shows significant potential to treat multi-drug resistant Pseudomonas aeruginosa infection in cystic fibrosis (CF), according to the case report of a CF patient with recurrent pneumonia and pulmonary exacerbations. These positive early results further support the development of the treatment against…

One year of treatment with Orkambi (lumacaftor/ivacaftor) returned blood glucose (sugar) levels to normal in almost half of a small group of diabetic or glucose-intolerant cystic fibrosis (CF) patients, a study reports. These results were summarized in the presentation “Observational study of glucose tolerance abnormalities in patients with cystic…

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…