ReviewEuropean journal of pediatrics2016
Searching for a cure for cystic fibrosis. A 25-year quest in a nutshell.
Review in European journal of pediatrics, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 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
22 citing papers in PubMed, 44 citations in OpenAlex.
- Ivacaftor Reduces Inflammatory Mediators in Upper Airway Lining Fluid From Cystic Fibrosis Patients With a G551D Mutation: Serial Non-Invasive Home-Based Collection of Upper Airway Lining Fluid.Frontiers in immunology · 2021Trial
- A novel treatment of cystic fibrosis acting on-target: cysteamine plus epigallocatechin gallate for the autophagy-dependent rescue of class II-mutated CFTR.Cell death and differentiation · 2016Trial
- A semi-automated algorithm for image analysis of respiratory organoids.PLoS computational biology · 2025Article
- Beyond the 10%: Unraveling the genetic diversity in Turkish cystic fibrosis patients not eligible for CFTR modulators.Pediatric pulmonology · 2024Article
- Theratyping of the Rare CFTR Genotype A559T in Rectal Organoids and Nasal Cells Reveals a Relevant Response to Elexacaftor (VX-445) and Tezacaftor (VX-661) Combination.International journal of molecular sciences · 2023Article
- A Cell-Based Optimised Approach for Rapid and Efficient Gene Editing of Human Pluripotent Stem Cells.International journal of molecular sciences · 2023Article
- Airway and Lung Organoids from Human-Induced Pluripotent Stem Cells Can Be Used to Assess CFTR Conductance.International journal of molecular sciences · 2023Article
- CFTR Modulator Therapy and Its Impact on Lung Transplantation in Cystic Fibrosis.Pulmonary therapy · 2021Review
- Evaluation of Fused Pyrrolothiazole Systems as Correctors of Mutant CFTR Protein.Molecules (Basel, Switzerland) · 2021Article
- Biosensors for Point Mutation Detection.Frontiers in bioengineering and biotechnology · 2021Article
- Precise Targeting of miRNA Sites Restores CFTR Activity in CF Bronchial Epithelial Cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2020Article
- Novel Human Polymorphisms Define a Key Role for the SLC26A6-STAS Domain in Protection From CaFrontiers in pharmacology · 2020Article
- Beyond cystic fibrosis transmembrane conductance regulator therapy: a perspective on gene therapy and small molecule treatment for cystic fibrosis.Gene therapy · 2019Review
- Pathogen acquisition in patients with cystic fibrosis receiving ivacaftor or lumacaftor/ivacaftor.Pediatric pulmonology · 2019Article
- Safety and efficacy of treatment with lumacaftor in combination with ivacaftor in younger patients with cystic fibrosis.Expert review of respiratory medicine · 2019Review
- Conformational change of the extracellular parts of the CFTR protein during channel gating.Cellular and molecular life sciences : CMLS · 2018Article
- TRPV6 Variants Interfere with Maternal-Fetal Calcium Transport through the Placenta and Cause Transient Neonatal Hyperparathyroidism.American journal of human genetics · 2018Article
- Pediatric lung transplantation.Journal of thoracic disease · 2017Review
- Medical and Surgical Advancements in the Management of Cystic Fibrosis Chronic Rhinosinusitis.Current otorhinolaryngology reports · 2017Article
- Can Cystic Fibrosis Patients Finally Catch a Breath With Lumacaftor/Ivacaftor?Clinical pharmacology and therapeutics · 2017Review
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 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
After 25 years of intensive search, there is not yet a cure for cystic fibrosis (CF). However, the quest has led to major breakthroughs in understanding the basic disease defect and defining strategies to correct it. The first cystic fibrosis transmembrane conductance regulator (CFTR) modulators have been introduced in clinic. Some show an impressive clinical benefit, like the potentiator ivacaftor for the 4% of patients with a class III defect. Others offer at present only a limited benefit, like the combination corrector lumacaftor plus potentiator ivacaftor for subjects homozygous for F508del. These findings prove that the basic defect in CF can be modified and hold the promise that one day CF will no longer be a life-shortening disease.
conclusionThis review updates the clinician on recent achievements as well as on the CF research pipeline. WHAT IS KNOWN: Cystic fibrosis (CF) is a common and life-shortening disease that currently cannot be cured. However, for each of the six CF mutation classes, disease-modifying drugs are under way. WHAT IS NEW: This review is a concise update for the clinician on new drugs that reached the CF clinical pipeline. The research strategies in CF have become a paradigm for clinical trials in other inherited diseases.
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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.