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Article

One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies.

Cells - 8 Jun 2022

Ensinck Marjolein M, Carlon Marianne S

Abstract excerpt

Cystic fibrosis (CF) is the most common monogenic disorder, caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Over the last 30 years, tremendous progress has been made in understanding the molecular basis of CF and the development of treatments that target the underlying defects in CF. Currently, a highly effective CFTR modulator treatment (Kalydeco™/Trikafta™) is available for 90% of...

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PMID: 35740997
DOI: 10.3390/cells11121868
PMCID: PMC9220995

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