cystic fibrosis

Low birth weight and poor weight gain in the first year of life in infants with cystic fibrosis (CF) predict poor growth rates due to pancreatic dysfunction, a study has found. The study, “Factors affecting the growth of infants diagnosed…

The Comité Économique des Produits de Santé (CEPS) in France has approved national reimbursement of Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) for patients with cystic fibrosis (CF) who are 2 years of age or older, with two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)…

The National Institutes of Health (NIH) has granted $2.7 million to Michigan State University (MSU) scientist Robert Quinn to investigate, in collaboration with Spectrum Health, the role of anaerobic bacteria in the development of flare-ups among patients with cystic fibrosis (CF). Anaerobic bacteria are a type…

Girls with cystic fibrosis-related diabetes (CFRD) are exposed to risk factors of early death — including chronic infections, poor lung function, and poor nutrition — at a significantly higher proportion and younger age than boys, a large European study shows. The findings highlight the importance of early diagnosis and effective treatment…

A new, more efficient method for delivering gene therapies to lung tissues — based on tiny engineered proteins, called peptides —is creating therapeutic opportunities for people with diseases like cystic fibrosis, chronic obstructive pulmonary disease or COPD, and asthma. The potential of these new delivery tools was described…

Pseudomonas aeruginosa, a bacteria that commonly causes serious lung infections in people with cystic fibrosis (CF), reacts to other dangerous bacteria in ways that could be exploited to develop new antimicrobial treatments, early research has found. When Staphylococcus aureus is present, P. aeruginosa starts moving…

People with cystic fibrosis (CF) show a “high adherence” to CFTR modulators, but these newer medications are costly and could undermine compliance to treatment over time, according to a joint studies by AllianceRx Walgreens Prime and Duquesne University School of Pharmacy. These findings were presented…

The Regional Pharmaceutical Procurement Service in Northern Ireland and the National Health Service (NHS) Wales have reached an agreement with Vertex Pharmaceuticals that will allow people with cystic fibrosis (CF) who live there to access all of Vertex’s currently approved medications for the disease. The terms of the…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…

Proteostasis Therapeutics (PTI) has completed patient enrollment in a Phase 2 clinical trial evaluating its cystic fibrosis transmembrane conductance regulator (CFTR) modulator combination therapies in cystic fibrosis (CF) patients who have at least one copy of the F508del mutation in the CFTR gene (the defective gene in CF…