ReviewPaediatric drugs2022
An Update on CFTR Modulators as New Therapies for Cystic Fibrosis.
Review in Paediatric drugs, 2022. 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 19 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.
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
19 citing papers in PubMed, 1 synthesis or guideline pooled it, 43 citations in OpenAlex.
- SMALL INTESTINAL BACTERIAL OVERGROWTH IN PEOPLE WITH CYSTIC FIBROSIS: SYSTEMATIC REVIEW.Arquivos de gastroenterologia · 2025Pooled it
- Article
- Review
- Cystic fibrosis transmembrane conductance regulator modulator therapy and lived experiences in South Africa: A mixed-methods study.The South African journal of physiotherapy · 2026Article
- Cystic Fibrosis and Primary Ciliary Dyskinesia Share Extensive Similarities: Is It Time to Place Them Under a Common Umbrella?Pediatric pulmonology · 2025Article
- Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis.Pulmonary therapy · 2025Review
- Strength-Oriented Virtual Exercise Training Intervention in Children and Adolescents with Cystic Fibrosis Under CFTR Modulators (the FIQMODE Study): Study Protocol for a Randomized Controlled Trial.Current protocols · 2025Article
- Dipeptidyl peptidase-1 inhibitors in bronchiectasis.European respiratory review : an official journal of the European Respiratory Society · 2025Review
- Clinical Outcomes in Patients with Cystic Fibrosis Receiving CFTR Modulators: A Comparison of Childhood Versus Adolescent Initiation.Children (Basel, Switzerland) · 2025Article
- Association Between Lung Parenchymal Attenuation in Computed Tomography and Airflow Limitation in Adults with Cystic Fibrosis.Diagnostics (Basel, Switzerland) · 2025Article
- Review
- Lung function improvement on triple modulators: high-resolution, nationwide data from the Danish Cystic Fibrosis Cohort.ERJ open research · 2024Article
- Affordable Cystic Fibrosis (CF) Transmembrane Conductance Regulator Modulator Drugs for CF: All CF Lives Worldwide Matter!American journal of respiratory and critical care medicine · 2023Article
- Air-Liquid interface cultures to model drug delivery through the mucociliary epithelial barrier.Advanced drug delivery reviews · 2023Review
- It Takes Two to Tango! Protein-Protein Interactions behind cAMP-Mediated CFTR Regulation.International journal of molecular sciences · 2023Review
- Improved Trimethylangelicin Analogs for Cystic Fibrosis: Design, Synthesis and Preliminary Screening.International journal of molecular sciences · 2022Article
- Cystic Fibrosis and Oxidative Stress: The Role of CFTR.Molecules (Basel, Switzerland) · 2022Review
- Therapeutic Drug Monitoring of Ivacaftor, Lumacaftor, Tezacaftor, and Elexacaftor in Cystic Fibrosis: Where Are We Now?Pharmaceutics · 2022Review
- Remotely addressing health-related social needs (ELICIT) in cystic fibrosis: design and implementation of a multicenter screening and intervention quality improvement project.Therapeutic advances in respiratory diseaseArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Over the past decade there have been significant developments in the field of Cystic Fibrosis Transmembrane Regulator modulator drugs. Following treatment in patients with cystic fibrosis with common gating mutations using the potentiator drug ivacaftor, successive development of corrector drugs used in combination has led to highly effective modulator therapy being available to more than 85% of the cystic fibrosis population over 12 years of age in the form of elexacaftor/tezacaftor/ivacaftor. In this article, we review the evidence from clinical trials and mounting real-world observational and registry data that demonstrates the impact highly effective modulators have on both pulmonary and extra-pulmonary manifestations of cystic fibrosis. As clinical trials progress to younger patient groups, we discuss the challenges to demonstrating drug efficacy in early life, and also consider practicalities of drug development in an ever-shrinking modulator-naïve population. Drug-drug interactions are an important consideration in people with cystic fibrosis, where polypharmacy is commonplace, but also as the modulated population look to remain healthier for longer, we identify trials that aim to address treatment burden too. Inequity of care, through drug cost or ineligibility for modulators by genotype, is widening without apparent strategies to address this; however, we present evidence of hopeful early-stage drug development for non-modulatable genes and summarise the current state of gene-therapy development.
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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.