Identifying the most effective drug combination for the treatment of Cystic Fibrosis
DOI:
https://doi.org/10.58445/rars.2155Keywords:
Cystic Fibrosis (CF), Cystic fibrosis transmembrane regulator (CFTR), Potentiator, Ivacaftor, CorrectorAbstract
Cystic fibrosis transmembrane regulator (CFTR) is a cell membrane protein that serves as a chloride ion channel. If defective CFTR protein is synthesized, it interferes with the function of the secreted mucus, changing its consistency. The most common cause of the defective CFTR protein is deletion mutation indicated as F508del. 80% of CF patients carry this mutation and suffer from the symptoms associated with thick mucus. Correctors are artificially designed drugs to minimize the effect of defective CFTR protein. Correctors are commonly used in combination with a potentiator, Ivacaftor. To date, three such corrector-potentiator combinations have been used, namely, LUMA/IVA, TEZA/IVA and ELEXA/TEZA/IVA (Trikafta). More than 85% of CF patients are subjected to this therapy. This paper reviews the effectiveness of these combinations, studying the parameters that indicate lung, liver, and pancreas functions.
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