Keywords
cystic fibrosis
Phe508del mitation
combination correctors
potentiators
Phe508del mitation
combination correctors
potentiators
How to Cite
Basta, K., & John, C. (2019). Combination Correctors and Potentiators for Cystic Fibrosis: A Systematic Review and Meta-analysis . The Physician, 6(1), c7. https://doi.org/10.38192/1.6.1.c7
Abstract
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.
References
Peer reviewed by Sunil Daga PhD MRCP and Subarna Chakravorty PhD FRCPath for BAPIO Annual Conference, London 2019
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