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Article

Recent advances in developing therapeutics for cystic fibrosis.

Human molecular genetics - 1 Aug 2018

Strug Lisa J, Stephenson Anne L, Panjwani Naim, Harris Ann

Abstract excerpt

Despite hope that a cure was imminent when the causative gene was cloned nearly 30 years ago, cystic fibrosis (CF [MIM: 219700]) remains a life-shortening disease affecting more than 70 000 individuals worldwide. However, within the last 6 years the Food and Drug Administration's approval of Ivacaftor, the first drug that corrects the defective cystic fibrosis transmembrane conductance regulator protein [CFTR...

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Identifiers

PMID: 30060192
DOI: 10.1093/hmg/ddy188
PMCID: PMC6061831

Genes

CFTR

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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