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Healthy kids help medical researchThat's why Melissa has signed up her four year old healthy son Odin for a study at Perth's The Kids Research Institute Australia that will help kids with cystic fibrosis.
News & Events
Prestigious award for cystic fibrosis researcherProfessor Stephen Stick has been awarded the prestigious Richard C. Talamo Distinguished Clinical Achievement Award from the US Cystic Fibrosis Foundation.
Research
Protocol for establishing a core outcome set for evaluation in studies of pulmonary exacerbations in people with cystic fibrosisPulmonary exacerbations are associated with increased morbidity and mortality in people with cystic fibrosis (CF). There is no consensus about which outcomes should be evaluated in studies of pulmonary exacerbations or how these outcomes should be measured.
Research
Pseudomonas aeruginosa modulates neutrophil granule exocytosis in an in vitro model of airway infectionA population of neutrophils recruited into cystic fibrosis (CF) airways is associated with proteolytic lung damage, exhibiting high expression of primary granule exocytosis marker CD63 and reduced phagocytic receptor CD16. Causative factors for this population are unknown, limiting intervention. Here we present a laboratory model to characterize responses of differentiated airway epithelium and neutrophils following respiratory infection.
Research
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway modelsMutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (CF), irrespective of their CF transmembrane conductance regulator (CFTR) gene variants. The aim of this study was to employ two CF rat models, Phe508del and CFTR knockout to assess the comparative effectiveness of CFTR modulators and lentiviral vector-mediated gene therapy.
COMBAT CF is one of two long-standing international trials which have resulted in new early intervention options helping to reduce progressive lung damage in kids living with CF.
Research
Substrate-dependent metabolomic signatures of myeloperoxidase activity in airway epithelial cells: Implications for early cystic fibrosis lung diseaseMyeloperoxidase is released by neutrophils in inflamed tissues. MPO oxidizes chloride, bromide, and thiocyanate to produce hypochlorous acid, hypobromous acid, and hypothiocyanous acid, respectively. These oxidants are toxic to pathogens, but may also react with host cells to elicit biological activity and potential toxicity. In cystic fibrosis and related diseases, increased neutrophil inflammation leads to increased airway MPO and airway epithelial cell exposure to its oxidants.
Research
Discrete choice experiment to evaluate preferences of patients with cystic fibrosis among alternative treatment-related health outcomes: A protocolThe aim of this study is to identify and quantify the preferences of patients with cystic fibrosis regarding treatment outcomes
Research
Trial refresh: A case for an adaptive platform trial for pulmonary exacerbations of cystic fibrosisThis review will discuss the necessary steps required for a Bayesian adaptive platform trial to optimize treatment of pulmonary exacerbations of cystic fibrosis
Research
Elastase Exocytosis by Airway Neutrophils Associates with Early Lung Damage in Cystic Fibrosis ChildrenProtease elastase exocytosis by airway neutrophils occurs in all cystic fibrosis children, and its cellular measure correlates with early lung damage