Optimalisatie van uitkomstparameters voor evaluatie van het effect van nieuwe pharmaca voor behandeling van mucoviscidose KU Leuven
Introduction Cystic Fibrosis Cystic Fibrosis (CF) is the most common severe hereditary disease in Caucasian populations, affecting approximately 1 in 3500 births. Mutations in the CFTR gene cause abnormal production and/or function of the CFTR protein, an anion channel in the apical membrane with a high expression in most cells lining the airways and the ducts of exocrine glands as the sweat gland, the pancreas or the vas deferens. The abnormal ...