ReviewCurrent opinion in pediatrics2018
Cystic fibrosis transmembrane conductance regulator modulators: precision medicine in cystic fibrosis.
Review in Current opinion in pediatrics, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06322446 (Effects of a Strength Exercise Program on Muscular Health in People With CF Treated With Modulators of the Cystic Fibrosis Transmembrane Conductance Regulator Receptor), which is not on this map. Cited by 32 papers, 2 of them syntheses 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.
Effects of a Strength Exercise Program on Muscular Health in People With CF Treated With Modulators of the Cystic Fibrosis Transmembrane Conductance Regulator Receptor (CFTR)
Who cites it
32 citing papers in PubMed, 2 syntheses or guidelines pooled it, 67 citations in OpenAlex.
- Efficacy and safety profile of elexacaftor-tezacaftor-ivacaftor triple therapy on cystic fibrosis: a systematic review and single arm meta-analysis.Frontiers in pharmacology · 2023Pooled it
- Effectiveness of positive expiratory pressure on patients over 16 years of age with cystic fibrosis: systematic review and meta-analysis.Therapeutic advances in respiratory diseasePooled it
- Discovery of Autophagy Modulators that Increase I1061T NPC1 Expression and Promote Cholesterol Efflux in Niemann-Pick Type C Patient-Derived Fibroblasts.ACS chemical biology · 2026Article
- A chronicInfection and immunity · 2025Article
- Effects of Elevated Glucose on Bacterial Respiratory Infections in Cystic Fibrosis and Chronic Airway Diseases.International journal of molecular sciences · 2025Review
- Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation.Children (Basel, Switzerland) · 2025Article
- Rare disease genomics and precision medicine.Genomics & informatics · 2024Review
- Therapeutic Interventions forJournal of clinical medicine · 2024Review
- Insights intoJournal of fungi (Basel, Switzerland) · 2024Article
- Chloride ions in health and disease.Bioscience reports · 2024Review
- Current landscape of cystic fibrosis gene therapy.Frontiers in pharmacology · 2024Review
- Review
- Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatment.Scientific reports · 2021Article
- Milestones of Precision Medicine: An Innovative, Multidisciplinary Overview.Molecular diagnosis & therapy · 2021Review
- Role of RND Efflux Pumps in Drug Resistance of Cystic Fibrosis Pathogens.Antibiotics (Basel, Switzerland) · 2021Review
- CFTR modulator therapy for cystic fibrosis caused by the rare c.3700A>G mutation.Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021Article
- Soft, skin-interfaced sweat stickers for cystic fibrosis diagnosis and management.Science translational medicine · 2021Article
- Article
- 1-BENZYLSPIRO[PIPERIDINE-4,1'-PYRIDO[3,4-b]indole] 'co-potentiators' for minimal function CFTR mutants.European journal of medicinal chemistry · 2021Article
- Enhancing Cystic Fibrosis Immune Regulation.Frontiers in pharmacology · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
purpose of reviewThe aim of this study was to describe the newest development in cystic fibrosis (CF) care, CF transmembrane conductance regulator (CFTR) modulator therapies. RECENT
findingsPhase II results showing CFTR modulator triple therapies are more effective than current CFTR modulators. SUMMARY: CFTR modulator therapy targets the protein defective in CF and boosts its function, but the drug must match mutation pathobiology. Ivacaftor, a CFTR potentiator, was the first modulator approved in 2012, with impressive improvement in lung function and other measures of disease in patients with gating and other residual function mutations (∼10% of CF patients). In 2015, the combination of lumacaftor, a CFTR corrector, and ivacaftor was approved for patients homozygous for the F508del mutation (∼40-50% of the CF population) with positive but less impressive clinical response and 10-20% incidence of intolerance. A next-generation CFTR corrector, tezacaftor, with ivacaftor equally effective and better tolerated than lumacaftor, has also received US Food and Drug Administration approval. Novel CFTR correctors, entering Phase 3 trials in triple modulator combination with tezacaftor-ivacaftor, appear substantially more effective for patients who are homozygous for the F508del mutation and can provide benefit for patients with a single F508del mutation. This offers promise of effective CFTR modulator therapy for nearly 90% of CF patients.
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.