Cystic fibrosis is a severe ion channel disease of autosomal recessive inheritance that is
caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene.
Thanks to continuously improved symptomatic treatment during the last five decades this lethal
paediatric disease has been transformed into a chronic disorder with a median life expectancy
of nowadays more than 50 years. This 2nd edition provides the reader with the background and
on-going preclinical and clinical research for the development of mutation-type specific
therapy of cystic fibrosis. Starting with the biology and biomarkers of CFTR in the context of
cystic fibrosis the reader gets insight into the basic and clinical research of CFTR
modulators from bench to bedside. A large section of the book focuses on the clinical trials
post-approval observational studies and the real-world experience with the CFTR modulators.
