Background Increasing the activity of defective cystic fibrosis transmembrane conductance
regulator (CFTR) protein is a potential treatment for cystic fibrosis. Methods We conducted a …

Background Cystic fibrosis is a life-limiting disease that is caused by defective or deficient
cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is the …

The past six decades have seen remarkable improvements in health outcomes for people
with cystic fibrosis, which was once a fatal disease of infants and young children. However, …

Inert gas washout tests, performed using the single- or multiple-breath washout technique,
were first described over 60 years ago. As measures of ventilation distribution inhomogeneity, …

Specialised CF care has led to a dramatic improvement in survival in CF: in the last four
decades, well above what was seen in the general population over the same period. With the …

Developments in managing CF continue to drive dramatic improvements in survival. As
newborn screening rolls-out across Europe, CF centres are increasingly caring for cohorts of …

Description: Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant disease
with an estimated prevalence of 1 in 5000 that is characterized by the presence of vascular …

The upregulation of nitric oxide (NO) by inflammatory cytokines and mediators in central and
peripheral airway sites can be monitored easily in exhaled air. It is now possible to estimate …

Cystic fibrosis (CF) is a fatal genetic disease caused by mutations in the CFTR (cystic fibrosis
transmembrane conductance regulator) gene, which regulates chloride and water transport …

Rationale: The cystic fibrosis (CF) modulator drug, elexacaftor/tezacaftor/ivacaftor (ETI), proved
highly effective in controlled clinical trials for individuals with at least one F508del allele, …