Introduction Cystic fibrosis (CF) is a multisystem disorder. Treatment is complex and
evidence for treatment decisions may be absent. Characterising gaps in the research …

The growing use of modulator therapies aimed at restoring cystic fibrosis transmembrane
conductance regulator (CFTR) protein function in people with cystic fibrosis has …

Introduction 'Highly effective'modulator therapies (HEMTs) have radically changed the Cystic
Fibrosis (CF) therapeutic landscape. These novel therapeutic approaches have permitted …

Background Although use of inhaled antibiotics is the standard of care in cystic fibrosis,
there is insufficient evidence to support use of inhaled antibiotics in patients with …

Rationale: Lung clearance index (LCI), measured by multiple breath washout (MBW), is a
noninvasive measure of ventilation inhomogeneity that holds promise as an objective …

Cystic fibrosis is the most common lethal inherited disease in Caucasians in the UK, with an
incidence of approximately one in 2500 live births. It is a heterogeneous disease which …

Background Maintenance of optimal lung function is an important therapeutic goal in cystic
fibrosis as it is lung damage that, in the long term, is responsible for most premature death …

There are a number of potential drugs for the treatment of cystic fibrosis (CF) currently
undergoing clinical studies. A number of antibacterials formulated for delivery by inhalation …

Background Ivacaftor has been shown to be a safe, effective treatment for cystic fibrosis in
patients aged 6 years or older with a CFTR gating mutation. We aimed to assess the safety …

Cystic fibrosis (CF) lung disease commences early in the disease progression and is the
most common cause of mortality. While new CF disease-modifying agents are currently …