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Interpretation of lung function in infants and young children with cystic fibrosis

This paper is a review of lung function tests best suited to use for early detection of lung disease in Cystic Fibrosis.

Multi-modality monitoring of cystic fibrosis lung disease: the role of chest computed tomography

Stratification of monitoring protocols based on the risk profile of the patient can help us in the future to better care for people with Cystic Fibrosis.

Assessment of early bronchiectasis in young children with cystic fibrosis is dependent on lung volume

The aim of this study was to determine whether assessment of early CT scan-detected bronchiectasis in young children with cystic fibrosis (CF) depends on...

Chest computed tomography: a validated surrogate endpoint of cystic fibrosis lung disease?

Clinical trials for the treatment of cystic fibrosis (CF) lung disease are important to test and optimise new therapeutic interventions.

Distribution of Early Structural Lung Changes due to Cystic Fibrosis Detected with Chest Computed Tomography

To examine the distribution of early structural lung changes in clinically stable infants and young children with cystic fibrosis using chest computed...

Novel end points for clinical trials in young children with cystic fibrosis

Cystic fibrosis (CF) lung disease commences early in the disease progression and is the most common cause of mortality.

SLC6A14 Impacts Cystic Fibrosis Lung Disease Severity via mTOR and Epithelial Repair Modulation

Cystic fibrosis (CF), due to pathogenic variants in CFTR gene, is associated with chronic infection/inflammation responsible for airway epithelium alteration and lung function decline. Modifier genes induce phenotype variability between people with CF (pwCF) carrying the same CFTR variants. Among these, the gene encoding for the amino acid transporter SLC6A14 has been associated with lung disease severity and age of primary airway infection by the bacteria Pseudomonas aeruginosa.

Investigating the Implications of CFTR Exon Skipping Using a Cftr Exon 9 Deleted Mouse Model

Severity and disease progression in people with Cystic Fibrosis is typically dependent on their genotype. One potential therapeutic strategy for people with specific mutations is exon skipping with antisense oligonucleotides. CFTR exon 9 is an in-frame exon and hence the exclusion of this exon would excise only 31 amino acids but not alter the reading frame of the remaining mRNA.

Preferred health outcome states following treatment for pulmonary exacerbations of cystic fibrosis

Treatment for pulmonary exacerbations of cystic fibrosis (CF) can produce a range of positive and negative outcomes. Understanding which of these outcomes are achievable and desirable to people affected by disease is critical to agreeing to goals of therapy and determining endpoints for trials.

Posaconazole-induced hypertension in children with cystic fibrosis

Posaconazole is a triazole antifungal with a broad spectrum of activity against moulds including Aspergillus spp. Emerging data suggest posaconazole may be effective in the treatment of allergic bronchopulmonary aspergillosis complicating cystic fibrosis.