text:"ystic fibrosis therapy means inevitably a complex and customized treatment plan for each patient. It consists of a range of components. These include medication such as CFTR modulators and antibiotics as well as inhalation therapy and mucolytics, physiotherapy, nutritional therapy and sports therapy. It is therefore essential that CF patients receive treatment at a specialist centre [1]. "
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introduction:"CFTR modulators represent a significant advancement in CF treatment since they are small molecules improving the function of the defective CFTR protein in a mutation-specific way, which helps restore chloride ion transport across cell membranes. Notable pharmaceutical agents include Trikafta®, Symdeko®, Orkambi® and Kalydeco® [1]. These medications have been demonstrated to significantly improve lung function and reduce pulmonary exacerbations. However, they are expensive and may cause side effects such as liver enzyme elevations and cataracts in pediatric patients [2]. Furthermore, they are not suitable for all CF patients since only mutations which produce a CFTR channel can be supported by CFTR modulators, not those mutations which lead to a missing CFTR channel (knock out) [1], e.g. stop-mutations including p.Arg553Ter or p.Gly542Ter [3]. ",