The most common cause of cystic fibrosis (CF) is the Phe508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, resulting in CFTR’s reduced trafficking, targeted by correctors, and reduced functioning, targeted by potentiators. For the majority of CF patients, the combination correctors and potentiators (CCPs) lumacaftor/ivacaftor and tezacaftor/ivacaftor, are the only treatments licensed to target the disease origin.
Peer reviewed by Sunil Daga PhD MRCP and Subarna Chakravorty PhD FRCPath for BAPIO Annual Conference, London 2019
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