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Among various lethal genetic syndromes, cystic fibrosis is one of them affecting the caucasian population. Ivacaftor has emerged as the first orally manageable drug that participated & succeeded in human clinical trials for cystic fibrosis sufferers. Ivacaftor operates as a cystic fibrosis transmembrane conductance regulator (CFTR) protein potentia...
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... duodenum, pancreas, lung, salivary glands and genital tracts. The viscous mucus secretions are injurious to the normal functioning of the above mentioned organs, thereby accountable for characteristic lung disease, pancreatic insufficiency, reduced intestinal absorption, reproductive dysfunction and high sweat chloride concentrations. [16,17] " Fig. 2" below depicts the pathophysiologic cascade of cystic fibrosis affecting the respiratory system. [19] www.wjpps.com Vol 5, Issue 11, 2016. 647 Another most widespread CF causing mutation is F508del-CFTR in which there is removal of amino acid phenylalanine at position 508 in the CFTR protein arrangement, leading to class II mutation ...
