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Edited by M.A. Mall and J.S. Elborn
Cystic fibrosis (CF) is one of the most common fatal hereditary diseases. The discovery of the CFTR gene 25 years ago set the stage for unraveling the pathogenesis of CF lung disease, continuous refinement of symptomatic treatments and the development of mutation-specific therapies, which are now becoming available for a subgroup of patients. This Monograph provides an update on all aspects of CF lung disease, from infancy to adulthood, including current concepts on the disease process, improvements in early diagnosis and monitoring, therapeutic approaches and patient care. In state-of the art chapters, this Monograph highlights important recent developments and discusses the next steps that will be required for further improvement of life expectancy and quality of life of patients with CF. It will be an essential reference for basic and clinical scientists and all members of the CF team.
"Engaging and enlightening [...] concise but rather comprehensive, and well-written. It offers a great deal to the experienced CF clinician and scientist, but is not so dense that it can also be recommended for the novice." Patrick Flume, Journal of Cystic Fibrosis
"...almost all physicians and even specialised care workers would profit from this issue. For the medical doctor caring daily for CF patients, it provides a short and concise update on current knowledge. For the senior researcher in the CF-research lab, it is a nice attractive book to be given to students and lab personnel who wish to get into the field of cystic fibrosis." Martin H. Schöni, European Journal of Pediatrics
1. Pathophysiology of cystic fibrosis lung disease
2. Airway inflammation in cystic fibrosis
3. Airway infection and the microbiome
4. Genetic and environmental modifiers of cystic fibrosis
5. Newborn screening for cystic fibrosis: opportunities and remaining challenges
6. Early cystic fibrosis lung disease
7. Imaging the lungs in cystic fibrosis
8. End-points and biomarkers for clinical trials in cystic fibrosis
9. Correcting the basic ion transport defects in cystic fibrosis
10. Potentiating and correcting mutant CFTR in patients
11. New horizons for cystic fibrosis gene and cell therapy
12. Improving airway clearance in cystic fibrosis lung disease
13. Antibiotic treatment of cystic fibrosis lung disease
14. Exercise in cystic fibrosis
15. Extrapulmonary manifestations of cystic fibrosis
16. Lung transplantation for cystic fibrosis
17. Standards of care for patients with cystic fibrosis
18. Using registries to improve cystic fibrosis care
19. Transition from paediatric to adult cystic fibrosis care: a developmental framework
20. Challenges of providing care to adults with cystic fibrosis
21. Health-economic aspects of cystic fibrosis screening and therapy
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