ReviewPediatric pulmonology2015
Breakthrough therapies: Cystic fibrosis (CF) potentiators and correctors.
Review in Pediatric pulmonology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 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
37 citing papers in PubMed.
- Elexacaftor/tezacaftor/ivacaftor in children aged ≥6 years with cystic fibrosis heterozygous forThe European respiratory journal · 2025Trial
- Transforming Care Models in Cystic Fibrosis: A Review.Healthcare (Basel, Switzerland) · 2025Review
- Special Issue "Research Advances on Cystic Fibrosis and CFTR Protein".International journal of molecular sciences · 2025Article
- AI-Driven Transcriptome Prediction in Human Pathology: From Molecular Insights to Clinical Applications.Biology · 2025Review
- Diagnosis and Management of Simple and Complicated Meconium Ileus in Cystic Fibrosis, a Systematic Review.Diagnostics (Basel, Switzerland) · 2024Review
- Overview of CFTR activators and their recent studies for dry eye disease: a review.RSC medicinal chemistry · 2023Review
- Cystin is required for maintaining fibrocystin (FPC) levels and safeguarding proteome integrity in mouse renal epithelial cells: A mechanistic connection between the kidney defects in cpk mice and human ARPKD.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2023Article
- Genome Editing for Cystic Fibrosis.Cells · 2023Review
- 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
- Effect of Trikafta on bone density, body composition and exercise capacity in CF: A pilot study.Pediatric pulmonology · 2023Article
- Clinical Phenotypes of Cystic Fibrosis Carriers.Annual review of medicine · 2022Review
- New drugs in cystic fibrosis: what has changed in the last decade?Therapeutic advances in chronic disease · 2022Review
- Review
- Safety and efficacy of the cystic fibrosis transmembrane conductance regulator potentiator icenticaftor (QBW251).Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society · 2021Article
- Cystic Fibrosis Point of Personalized Detection (CFPOPD): An Interactive Web Application.JMIR medical informatics · 2020Article
- The Pathophysiology of Rett Syndrome With a Focus on Breathing Dysfunctions.Physiology (Bethesda, Md.) · 2020Review
- Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells.Nucleic acids research · 2020Article
- CFTR targeted therapies: recent advances in cystic fibrosis and possibilities in other diseases of the airways.European respiratory review : an official journal of the European Respiratory Society · 2020Review
- Coordinate regulation of ELF5 and EHF at the chr11p13 CF modifier region.Journal of cellular and molecular medicine · 2019Article
- Ivacaftor improves rhinologic, psychologic, and sleep-related quality of life in G551D cystic fibrosis patients.International forum of allergy & rhinology · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
Cystic Fibrosis is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene resulting in abnormal protein function. Recent advances of targeted molecular therapies and high throughput screening have resulted in multiple drug therapies that target many important mutations in the CFTR protein. In this review, we provide the latest results and current progress of CFTR modulators for the treatment of cystic fibrosis, focusing on potentiators of CFTR channel gating and Phe508del processing correctors for the Phe508del CFTR mutation. Special emphasis is placed on the molecular basis underlying these new therapies and emerging results from the latest clinical trials. The future directions for augmenting the rescue of Phe508del with CFTR modulators are also emphasized.
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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.