Cystic Fibrosis

Microbiology of airway disease in a cohort of patients with Cystic Fibrosis
Recent reports document an increasing incidence of new Gram-negative pathogens such as Stenotrophomonas maltophilia and Alcaligenes xylosoxidans isolated from patients with Cystic Fibrosis, along with an increase in common Gram-negative pathogens such as Pseudomonas aeruginosa and Burkholderia cepacia complex.

The effect of inpatient rehabilitation programmes on quality of life in patients with cystic fibrosis: A multi-center study
Disease-specific, multimodal inpatient rehabilitation programmes are designed to improve the physical, emotional, and social functioning of patients with cystic fibrosis (CF).

The GCC repeat length in the 5'UTR of MRP1 gene is polymorphic: a functional characterization of its relevance for cystic fibrosis
Among the members of the ATP binding cassette transporter superfamily, MRPs share the closest homology with the CFTR protein, which is defective in CF disease. MRP1 has been proposed as a potential modifier gene and/or as novel target for pharmacotherapy of CF to explain the clinical benefits observed in some CF patients treated with the macrolide AZM.

In some patients with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. PTC124 has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether PTC124 can safely increase functional CFTR protein in the cells of patients with CF due to a nonsense mutation.

• Pharmacogenomics of the cystic fibrosis transmembrane conductance regulator (CFTR) and the cystic fibrosis drug CPX using genome microarray analysis
• Sildenafil (Viagra) corrects DeltaF508-CFTR location in nasal epithelial cells from patients with cystic fibrosis
• Pharmacotherapy of the ion transport defect in cystic fibrosis: role of purinergic receptor agonists and other potential therapeutics
• Pharmacological treatment of the ion transport defect in cystic fibrosis
• A novel natural product compound enhances cAMP-regulated chloride conductance of cells expressing CFTR[delta]F508

Acute volume and electrolyte replacement corrects hypochloremic hypovolemic metabolic alkalosis and compensatory hypoventilation/ hypercapnia in acute exacerbations of cystic fibrosis.

• Bone loss in patients with untreated chronic obstructive pulmonary disease is mediated by an increase in bone resorption associated with hypercapnia
• Effects of acetazolamide and furosemide on ventilation and cerebral blood volume in normocapnic and hypercapnic patients with COPD
• Re-evaluation of acid-base prediction rules in patients with chronic respiratory acidosis
• Metabolic alkalosis and cystic fibrosis
• Chronic metabolic alkalosis: not uncommon in young children with severe cystic fibrosis

This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.

• Predicting response to rhDNase and hypertonic saline in children with cystic fibrosis
• Aerosolized dornase alfa (rhDNase) for therapy of cystic fibrosis
• Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group
• Aerosolized recombinant human DNase I for the treatment of cystic fibrosis
• Comparison of hypertonic saline and alternate-day or daily recombinant human deoxyribonuclease in children with cystic fibrosis: a randomised trial