ArticleEMBO molecular medicine2017
Orkambi® and amplifier co-therapy improves function from a rare
Abstract read
In one paragraph
Article in EMBO molecular medicine, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 68 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
68citing papers in PubMed, 2 pooled it
–field-weighted citation impact
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
68 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- From Ivacaftor to Triple Combination: A Systematic Review of Efficacy and Safety of CFTR Modulators in People with Cystic Fibrosis.International journal of molecular sciences · 2020Pooled it
- A Systematic Review of the Clinical Efficacy and Safety of CFTR Modulators in Cystic Fibrosis.Scientific reports · 2019Pooled it
- Targeted Therapy for Restoring CFTR Activity: From Experimental to Clinical Features.International journal of molecular sciences · 2026Review
- Functional, Cohort-Level Assessment of CFTR Modulator Responses Using Biobanked Nasal Epithelial Cells from Individuals with Cystic Fibrosis.Journal of personalized medicine · 2026Article
- Review
- Therapeutic strategies to reverse cigarette smoke-induced ion channel and mucociliary dysfunction in COPD airway epithelial cells.American journal of physiology. Lung cellular and molecular physiology · 2025Article
- Nasal cells as a bronchial cell surrogate for pre-clinical assessment of drug response in cystic fibrosis.Frontiers in pharmacology · 2025Article
- Cystic Fibrosis Modulator Therapies: Bridging Insights from CF to other Membrane Protein Misfolding Diseases.Israel journal of chemistry · 2024Article
- Understanding CFTR Functionality: A Comprehensive Review of Tests and Modulator Therapy in Cystic Fibrosis.Cell biochemistry and biophysics · 2024Review
- Validating organoid-derived human intestinal monolayers for personalized therapy in cystic fibrosis.Life science alliance · 2023Article
- Mutation-class dependent signatures outweigh disease-associated processes in cystic fibrosis cells.Cell & bioscience · 2023Article
- Features of CFTR mRNA and implications for therapeutics development.Frontiers in genetics · 2023Review
- Workshop on RanBP2/Nup358 and acute necrotizing encephalopathy.Nucleus (Austin, Tex.) · 2022Article
- The CFTR Amplifier Nesolicaftor Rescues TGF-β1 Inhibition of Modulator-Corrected F508del CFTR Function.International journal of molecular sciences · 2022Article
- Molecular Dynamics and Theratyping in Airway and Gut Organoids Reveal R352Q-CFTR Conductance Defect.American journal of respiratory cell and molecular biology · 2022Article
- One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies.Cells · 2022Review
- Distinct proteostasis states drive pharmacologic chaperone susceptibility for cystic fibrosis transmembrane conductance regulator misfolding mutants.Molecular biology of the cell · 2022Article
- Proteostasis Regulators in Cystic Fibrosis: Current Development and Future Perspectives.Journal of medicinal chemistry · 2022Review
- Anticipating New Treatments for Cystic Fibrosis: A Global Survey of Researchers.Journal of clinical medicine · 2022Article
- CFTR interactome mapping using the mammalian membrane two-hybrid high-throughput screening system.Molecular systems biology · 2022Article
8 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
21 authors.
Steven V MolinskiDepartment of Biochemistry, University of Toronto, Toronto, ON, Canada.ORCID 0000-0001-9215-8232
Saumel AhmadiProgramme in Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0002-5800-6945
Wan IpProgramme in Translational Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.
Hong OuyangProgramme in Translational Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.
Adriana VillellaProteostasis Therapeutics, Cambridge, MA, USA.
John P MillerProteostasis Therapeutics, Cambridge, MA, USA.ORCID 0000-0003-1493-1394
Po-Shun LeeProteostasis Therapeutics, Cambridge, MA, USA.
Kethika KulleperumaDepartment of Biochemistry, University of Toronto, Toronto, ON, Canada.
Kai DuProgramme in Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.
Michelle Di PaolaDepartment of Biochemistry, University of Toronto, Toronto, ON, Canada.
Paul Dw EckfordProgramme in Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.
Onofrio LaselvaProgramme in Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0002-0237-4079
Ling Jun HuanProgramme in Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.
Leigh WellhauserProgramme in Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0003-0883-1768
Ellen LiProgramme in Molecular Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.
Peter N RayDivision of Molecular Genetics, Hospital for Sick Children, Toronto, ON, Canada.
Régis PomèsDepartment of Biochemistry, University of Toronto, Toronto, ON, Canada.
Theo J MoraesProgramme in Translational Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0001-9968-6601
Tanja GonskaProgramme in Translational Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0001-6786-4354
Felix RatjenDivision of Respiratory Medicine, Hospital for Sick Children, Toronto, ON, Canada.ORCID 0000-0003-4057-6592
Christine E BearDepartment of Biochemistry, University of Toronto, Toronto, ON, Canada bear@sickkids.ca.ORCID 0000-0001-7063-3418
Funding
CIHR GPG‐102171CIHR MOP‐130461CIHR MOP‐97954
Abstract
The combination therapy of lumacaftor and ivacaftor (Orkambi
Indexed as
Genetic TherapyAminophenolsAminopyridinesBenzodioxolesCombined Modality TherapyCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDrug CombinationsGene EditingHumansPoint MutationQuinolonesAminophenolsAminopyridinesBenzodioxolesCystic Fibrosis Transmembrane Conductance RegulatorDrug Combinationslumacaftor, ivacaftor drug combinationQuinolonesamplifierc.3700 A>GCFTRCRISPR/Cas9cystic fibrosis
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY
Read underepoch 390
Registered trials
None linked
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.