A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.
Ivacaftor was associated with improvements in lung function at 2 weeks that were sustained through 48 weeks and substantial improvements were also observed in the risk of pulmonary exacerbations, patient-reported respiratory symptoms, weight, and concentration of sweat chloride.
Allergic bronchopulmonary aspergillosis in cystic fibrosis--state of the art: Cystic Fibrosis Foundation Consensus Conference.
Virulence factors in Aspergillus that could aggravate these diseases, and particularly immunogenetic factors that could predispose persons to ABPA, were identified and diagnostic criteria that could provide a framework for monitoring were adopted.
Fungi and allergic lower respiratory tract diseases.
Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation
In this study, VX-809 had a similar adverse event profile to placebo for 28 days in F508del-CFTR homozygous patients, and demonstrated biological activity with positive impact on CFTR function in the sweat gland.
Late pulmonary sequelae of bronchopulmonary dysplasia.
Most adolescents and young adults who had bronchopulmonary dysplasia in infancy have some degree of pulmonary dysfunction, consisting of airway obstruction, airway hyperreactivity, and hyperinflation.
Sputum cathelicidin, urokinase plasminogen activation system components, and cytokines discriminate cystic fibrosis, COPD, and asthma inflammation.
Induced-sputum innate immune factor levels discriminate inflammatory changes in CF, COPD, and asthma, suggesting potential roles in pathophysiology and as well as providing disease-specific biomarker patterns.
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a…
Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation.
This study showed that VX-770 was associated with within-subject improvements in CFTR and lung function and provides support for further studies of pharmacologic potentiation of CFTR as a means to treat cystic fibrosis.
Significant microbiological effect of inhaled tobramycin in young children with cystic fibrosis.
- R. Gibson, J. Emerson, B. Ramsey
- MedicineAmerican Journal of Respiratory and Critical Care…
- 15 March 2003
It is concluded that 28 days of tobramycin solution for inhalation of 300 mg twice daily is safe and effective for significant reduction of lower airway Pa density in young children with cystic fibrosis.
Pulmonary sequelae of bronchopulmonary dysplasia survivors: high-resolution CT findings.
- S. Howling, W. Northway, D. Hansell, R. Moss, S. Ward, N. Müller
- MedicineAJR. American journal of roentgenology
- 1 May 2000
The cardinal CT features of bronchopulmonary dysplasia survivors include multifocal areas of reduced lung attenuation and perfusion, bronchial wall thickening, and decreased bronchus-to-pulmonary artery diameter ratios.