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

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, …

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 …

The views and recommendations made in this document do not represent the official position
of any publisher or professional medical society. This document has not been endorsed by …

Lung infections with Mycobacterium abscessus, a species of multidrug-resistant nontuberculous
mycobacteria, are emerging as an important global threat to individuals with cystic …

Background The phe508del CFTR mutation causes cystic fibrosis by limiting the amount of
CFTR protein that reaches the epithelial cell surface. We tested combination treatment with …

… Genetic analyses of the initial cohort showed that a single copy of the SERPINA1 Z allele
and each additional copy of the TGFB1 codon 10 C allele were associated with significantly …

The incidence and number of deaths from non-tuberculous mycobacterial (NTM) disease
have been steadily increasing globally. These lesser known “cousins” of Mycobacterium …

Prediction models aim to use available data to predict a health state or outcome that has not
yet been observed. Prediction is primarily relevant to clinical practice, but is also used in …