News

Vertex Pharmaceuticals’ treatments for cystic fibrosis (CF) all provide considerable benefits, but their pricing is not transparent and does not align with the medications clinical value, a review of the Institute for Clinical and Economic Review (ICER)’s report by the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC)…

New research provides insights into how bacteria “talk” to each other in lung infections, which could provide strategies to block these signals and ease bacterial virulence in patients with cystic fibrosis (CF). The study, “Spatial determinants of quorum signaling in a Pseudomonas aeruginosa infection model,” was…

Combining the approved multiple sclerosis treatment glatiramer acetate with the antibiotic tobramycin improved its ability to kill antibiotic-resistant strains of Pseudomonas aeruginosa isolated from cystic fibrosis (CF) patients, new data show. These results were shared in the presentation “The repurposed multiple sclerosis drug, glatiramer acetate, is an antibiotic resistance breaker in Pseudomonas aeruginosa…

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…