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The effects of cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators on lung function, pulmonary exacerbations, and quality of life have been well documented.
- Valentine Sergeev, Frank Y Chou, Grace Y Lam, Grace Y Lam, Christopher Michael Hamilton, Pearce G Wi...
- 2020
Oct 31, 2019 · Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the ...
- Peter G. Middleton, Marcus A. Mall, Pavel Dřevínek, Larry C. Lands, Edward F. McKone, Deepika Poline...
- 2019
The development of CFTR modulators has been one of the most remarkable stories in respiratory medicine. Defining the genetic, molecular, and cellular biology of cystic fibrosis (CF) mutations enabled high-throughput screening to identify compounds that partially restore CFTR function.
- Mike Myerburg, Joseph M. Pilewski
- 2021
Sep 30, 2023 · Emerging gene therapies for cystic fibrosis include inhaled delivery of CFTR mRNA in lipid nanoparticles or CFTR cDNA in viral vectors as mutation-agnostic approaches to restore
The success of highly effective modulator therapy (HEMT) in cystic fibrosis (CF) now illustrates two areas of deficiency: the lack of HEMT for younger children and for approximately 10% of the CF population without a qualifying mutation.
- Cori L. Daines, Wayne J. Morgan
- 2021
May 10, 2019 · This systematic review examines the clinical efficacy and safety of CFTR modulators in individuals with cystic fibrosis (CF) with specific genetic mutations.
People also ask
Are CFTR modulators effective in cystic fibrosis?
Does triple-combination CFTR modulator therapy work in patients with cystic fibrosis?
Are CFTR modulators a multiorgan disease?
What makes CFTR a success?
Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study.
