#CYSTIC FIBROSIS LUNG SOUNDS TRIAL#

Late diagnosis defines a unique population of long-term survivors of cystic fibrosis. Factors associated with FEV1 decline in cystic fibrosis: analysis of the ECFS patient registry. Canadian cystic fibrosis patient data registry report 2010. References 15 and 17 show the improvements in survival in the US and the UK cystic fibrosis populations, respectively, over the past decade.Ĭystic Fibrosis Canada. Cystic fibrosis mortality and survival in the UK: 1947–2003. (Cystic Fibrosis Foundation, 2013).ĭodge, J. 161, 233–241 (2014).Ĭystic Fibrosis Foundation Cystic Fibrosis Foundation Patient Registry 2012 annual data report. Longevity of patients with cystic fibrosis in 2000 to 2010 and beyond: survival analysis of the cystic fibrosis foundation patient registry. Improved survival among young patients with cystic fibrosis. Cystic fibrosis birth rates in Canada: a decreasing trend since the onset of genetic testing. High incidence of cystic fibrosis in children born in Italy to Albanian immigrants. Incidence of cystic fibrosis in the Albanian population. The prevalence of cystic fibrosis in the European Union. in Hodson and Geddes' Cystic Fibrosis 3rd edn (eds Hodson, M. Identification of the cystic fibrosis gene: chromosome walking and jumping. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Mutations in the cystic fibrosis transmembrane regulator gene and in vivo transepithelial potentials. Role of cystic fibrosis transmembrane conductance regulator in patients with chronic sinopulmonary disease. Cystic fibrosis transmembrane conductance regulator and obstructive azoospermia. Genetic influences on cystic fibrosis lung disease severity. Lastly, we discuss how cystic fibrosis care and treatment is expected to change in future years.

We also describe the epidemiology of cystic fibrosis and how cystic fibrosis and its treatment affect patients' quality of life.

In this Primer, we summarize the current understanding about how CFTR mutations cause disease and we outline current diagnostic strategies and therapeutic interventions addressing disease manifestations. Although these advances can mainly be attributed to improvements in symptom-directed therapy of downstream complications, it is expected that upstream CFTR-directed therapy will further improve patients' longevity and quality of life. Strong partnerships have also been developed between government agencies, academic centres, voluntary health organizations, individuals and families with cystic fibrosis.

#CYSTIC FIBROSIS LUNG SOUNDS TRIAL#

The development of clinical trial networks has enabled rapid testing of putative treatments through training of research staff, establishment of standard operating procedures and reference laboratories. Important advances in addressing all aspects of the disease have been made over the past two decades and the prognosis of patients with cystic fibrosis is constantly improving. The discovery of the cystic fibrosis gene defect in 1989 has resulted in a better understanding of disease pathophysiology, but only in the past few years has this information led to targeted therapies that address the underlying cellular defect 5– 7. For an illustrated summary of this Primer, visit: In this Primer, we summarize the current knowledge, and provide an outlook on how cystic fibrosis clinical care and research will be affected by new knowledge and therapeutic options in the near future. Until recently, the standard of care in cystic fibrosis treatment focused on preventing and treating complications of the disease now, novel treatment strategies directly targeting the ion channel abnormality are becoming available and it will be important to evaluate how these treatments affect disease progression and the quality of life of patients. Prenatal diagnostics, newborn screening and new treatment algorithms are changing the incidence and the prevalence of the disease. CFTR dysfunction affects many organs however, lung disease is responsible for the vast majority of morbidity and mortality in patients with cystic fibrosis. CFTR is a transmembrane protein that transports ions across the surface of epithelial cells. The gene defect was first described 25 years ago and much progress has been made since then in our understanding of how CFTR mutations cause disease and how this can be addressed therapeutically. Cystic fibrosis is an autosomal recessive, monogenetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR) gene.

Cystic fibrosis lung sounds