ReviewPediatric pulmonology2018
Rapid therapeutic advances in CFTR modulator science.
Review in Pediatric pulmonology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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
20 citing papers in PubMed, 52 citations in OpenAlex.
- Ivacaftor in People with Cystic Fibrosis and aAnnals of the American Thoracic Society · 2021Trial
- Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial.Annals of the American Thoracic Society · 2021Trial
- Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.The New England journal of medicine · 2019Trial
- Personalized tobramycin dosing in children with cystic fibrosis: an AUCAntimicrobial agents and chemotherapy · 2025Observational
- Article
- Real-world outcomes and direct care cost before and after elexacaftor/tezacaftor/ivacaftor initiation in commercially insured members with cystic fibrosis.Journal of managed care & specialty pharmacy · 2023Article
- Rate of Lung Function Decline in People with Cystic Fibrosis Having a Residual Function Gene Mutation.Pulmonary therapy · 2022Article
- Disease burden in people with cystic fibrosis heterozygous for F508del and a minimal function mutation.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2022Observational
- Mucosal Immunity in Cystic Fibrosis.Journal of immunology (Baltimore, Md. : 1950) · 2021Review
- Burden of cystic fibrosis in children <12 years of age prior to the introduction of CFTR modulator therapies.BMJ open respiratory research · 2021Article
- Inflammatory cytokines TNF-α and IL-17 enhance the efficacy of cystic fibrosis transmembrane conductance regulator modulators.The Journal of clinical investigation · 2021Article
- Peak glucose during an oral glucose tolerance test is associated with future diabetes risk in adults with cystic fibrosis.Diabetologia · 2021Article
- First clinical trials of the inhaled epithelial sodium channel inhibitor BI 1265162 in healthy volunteers.ERJ open research · 2021Article
- Article
- Novel therapeutic approaches for the management of cystic fibrosis.Multidisciplinary respiratory medicine · 2020Article
- Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants.Scientific reports · 2019Article
- Validity and Reliability of a Novel Multimodal Questionnaire for the Assessment of Abdominal Symptoms in People with Cystic Fibrosis (CFAbd-Score).The patient · 2019Article
- Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or twoERJ open research · 2019Article
- Is cellular senescence involved in cystic fibrosis?Respiratory research · 2019Review
- Rapid therapeutic advances in CFTR modulator science.Pediatric pulmonology · 2018Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Cystic fibrosis (CF) is an autosomal recessive genetic disease caused by variants in the gene encoding the cystic fibrosis transmembrane conduction regulator (CFTR) protein. Loss of CFTR function disrupts chloride, bicarbonate and regulation of sodium transport, producing a cascade of mucus obstruction, inflammation, pulmonary infection, and ultimately damage in numerous organs. Established CF therapies treat the downstream consequences of CFTR dysfunction and have led to steady improvements in patient survival. A class of drugs termed CFTR modulators has recently entered the CF therapeutic landscape. These drugs differ fundamentally from prior therapies in that they aim to improve the function of disease-causing CFTR variants. This review summarizes the science behind CFTR modulators, including their targets, mechanism of action, clinical benefit, and future directions in the field. CFTR modulators have dramatically changed how CF is treated, validated CFTR as a therapeutic target, and opened the door to truly personalized therapies and treatment regimens.
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.