ReviewLancet (London, England)2023
CFTR modulator therapy: transforming the landscape of clinical care in cystic fibrosis.
Review in Lancet (London, England), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 84 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
84 citing papers in PubMed, 1 synthesis or guideline pooled it, 135 citations in OpenAlex.
- CFTR modulator monotherapy for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).The Cochrane database of systematic reviews · 2026Pooled it
- Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adolescents and adults with cystic fibrosis and F508del-gating and F508del-residual function genotypes: results from an open-label extension study.Annals of the American Thoracic Society · 2026Trial
- Risk Carriers: Elevated Risk of Post-ERCP Pancreatitis in Patients Heterozygous for Mutations of the Cystic Firbrosis Transmembrane Regulator (CFTR) Gene.Digestive diseases and sciences · 2026Article
- Kidney calcifications and fluid-electrolyte imbalances in cystic fibrosis: a simplified synopsis.Pediatric nephrology (Berlin, Germany) · 2026Article
- The dipeptidyl peptidase 1 inhibition landscape in bronchiectasis and its future direction.European respiratory review : an official journal of the European Respiratory Society · 2026Review
- Pseudomonas aeruginosa induces the generation of pathogenic IFNγNature microbiology · 2026Article
- Bridging the Gap in Eastern European Cystic Fibrosis Care: How Newborn Screening and Advanced CFTR Modulation Shape the Clinical Landscape in Western Romania.Children (Basel, Switzerland) · 2026Article
- Impact of air pollution on lung function in cystic fibrosis over a decade in London: a UK CF Registry study.Thorax · 2026Article
- Cystic Fibrosis Mortality Trends 1999-2024-A CDC Wonder Study.Advances in respiratory medicine · 2026Article
- Epithelial plasma membrane transporters as drug targets.Physiological reviews · 2026Review
- Maternal and fetal outcomes associated with CFTR modulator use during pregnancy: a scoping review.European respiratory review : an official journal of the European Respiratory Society · 2026Article
- Prenatal CFTR modulator therapy for fetal cystic fibrosis: Emerging evidence, clinical considerations, and future directions.Pregnancy (Hoboken, N.J.) · 2026Review
- Review
- Remodeling the pathological airway: advanced nanotechnology for diagnostics and therapeutics in cystic fibrosis.Journal of nanobiotechnology · 2026Review
- Characterization of two ultra-rare CFTR variants, P.Leu999del and P.Glu1104Lys, with unknown theratyping profiles.Orphanet journal of rare diseases · 2026Article
- Synaptic Plasticity-Intrinsic Excitability and Antidepressant Discovery.Biomedicines · 2026Review
- Elexacaftor/tezacaftor/ivacaftor is associated with long-term reduction in use of chronic respiratory therapies in cystic fibrosis.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2026Observational
- Non-viral delivery of genome-editing tools for treatment of genetic disorders.Acta pharmaceutica Sinica. B · 2026Review
- Psychological impact of genetic eligibility for CFTR modulator therapy in children with cystic fibrosis and their parents.European journal of pediatrics · 2026Article
- Single-molecule dissection of CFTR folding defects and pharmacological rescue.Proceedings of the National Academy of Sciences of the United States of America · 2026Article
24 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
4 authors at 4 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Following discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989 and subsequent elucidation of the varied CFTR protein abnormalities that result, a new era of cystic fibrosis management has emerged-one in which scientific principles translated from the bench to the bedside have enabled us to potentially treat the basic defect in the majority of children and adults with cystic fibrosis, with a resultant burgeoning adult cystic fibrosis population. However, the long-term effects of these therapies on the multiple manifestations of cystic fibrosis are still under investigation. Understanding the effects of modulators in populations excluded from clinical trials is also crucial. Furthermore, establishing appropriate disease measures to assess efficacy in the youngest potential trial participants and in those whose post-modulator lung function is in the typical range for people without chronic lung disease is essential for continued drug development. Finally, recognising that a health outcome gap has been created for some people and widened for others who are not eligible for, cannot tolerate, or do not have access to modulators is important.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.