The therapeutic triple combo elexacaftor (VX-445), tezacaftor, and ivacaftor leads to marked improvements in lung function and quality of life in people with cystic fibrosis (CF) carrying the F508del mutation, data from a Phase 3 trial show. The findings were reported in a study, “Efficacy and…
When it comes to lung transplants, the Cystic Fibrosis Foundation (CFF) admits that for a long time the issue was shrouded in confusion, frustration, and lack of information. But that’s changed substantially in recent years, says Albert Faro, MD, head of the nonprofit’s Advanced Lung Disease Initiative. “In…
Measuring the levels of insulin-like growth factor 2 receptor (IGF2R) during the first days of treatment with intravenous antibiotics could help predict treatment response in cystic fibrosis patients experiencing pulmonary exacerbations, a study has found. The study, “Identification of novel blood biomarkers of treatment response…
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…
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