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A CFTR potentiator in patients with cystic fibrosis and the G551D mutation.
TLDR
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.
TLDR
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.
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
TLDR
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.
Fungi and allergic lower respiratory tract diseases.
TLDR
The term severe asthma associated with fungal sensitization (SAFS) has been coined to illustrate the high rate of fungal sensitivity in patients with persistent severe asthma and improvement with antifungal treatment.
Late pulmonary sequelae of bronchopulmonary dysplasia.
TLDR
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.
TLDR
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.
Significant microbiological effect of inhaled tobramycin in young children with cystic fibrosis.
TLDR
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.
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
TLDR
The annualised rate of ppFEV1 decline was reduced in lumacaftor/ivacaftar-treated patients compared with matched controls, and the estimated annual rate of decline in percent predicted FEV1 in treated patients was compared with that of a matched registry cohort.
Pulmonary sequelae of bronchopulmonary dysplasia survivors: high-resolution CT findings.
TLDR
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.
Correlates of osteopenia in patients with cystic fibrosis.
TLDR
Osteopenia is common at all ages in children and adults with cystic fibrosis, suggesting that inadequate bone mineral accretion as well as increased bone loss contribute to the deficits in bone mineral observed.
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