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References from Long-term effectiveness and safety of cystic fibrosis transmembrane conductance regulator modulators and their impact on the lives of people with cystic fibrosis. Local targets link to admitted publications; unresolved targets remain external evidence.
Effects of elexacaftor/tezacaftor/ivacaftor therapy on CFTR function in patients with cystic fibrosis and one or two F508del alleles
10.1164/rccm.202110-2249oc · 2022 · External reference
Clinical mechanism of the cystic fibrosis transmembrane conductance regulator potentiator ivacaftor in G551D-mediated cystic fibrosis
10.1164/rccm.201404-0703oc · 2014 · External reference
Clinical effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: a clinical trial
10.1164/rccm.202108-1986oc · 2022 · External reference
Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in adults and adolescents with cystic fibrosis and at least one F508del allele: a phase 3, open-label extension study
10.1164/rccm.202411-2231oc · 2025 · External reference
The future of cystic fibrosis care: a global perspective
10.1016/s2213-2600(19)30337-6 · 2020 · External reference
Cystic fibrosis: emergence of highly effective targeted therapeutics and potential clinical implications
10.1164/rccm.201910-1943so · 2020 · External reference
A CFTR potentiator in patients with cystic fibrosis and the G551DMutation
10.1056/nejmoa1105185 · 2011 · External reference
Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation
10.1378/chest.11-2672 · 2012 · External reference
Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR
10.1056/nejmc1510466 · 2015 · External reference
Tezacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del
10.1056/nejmoa1709846 · 2017 · External reference
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial
10.1016/s0140-6736(19)32597-8 · 2019 · External reference
Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele
10.1056/nejmoa1908639 · 2019 · External reference
Efficacy and safety of elexacaftor/tezacaftor/ivacaftor in children 6 through 11 years of age with cystic fibrosis heterozygous for F508del and a minimal function mutation: a phase 3b, randomized, placebo-controlled study
10.1164/rccm.202202-0392oc · 2022 · External reference
Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials
10.1016/s2213-2600(24)00411-9 · 2025 · External reference
Real-world disparities and ethical considerations with access to CFTR modulator drugs: mind the gap!
10.3389/fphar.2023.1163391 · 2023 · External reference
Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation
10.1016/j.jcf.2020.11.008 · 2021 · External reference
Rapid improvement after starting elexacaftor-tezacaftor-ivacaftor in patients with cystic fibrosis and advanced pulmonary disease
10.1164/rccm.202011-4153oc · 2021 · External reference
Multisystemic effects of elexacaftor-tezacaftor-ivacaftor in adults with cystic fibrosis and advanced lung disease
10.1513/annalsats.202312-1065oc · 2024 · External reference
Impact of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in children with cystic fibrosis and one or two F508del alleles
10.1183/13993003.00004-2024 · 2024 · External reference
Longitudinal effects of elexacaftor/tezacaftor/ivacaftor on sputum viscoelastic properties, airway infection and inflammation in patients with cystic fibrosis
10.1183/13993003.02153-2022 · 2023 · External reference
Elexacaftor/tezacaftor/ivacaftor’s effects on cystic fibrosis infections are maintained, but not increased, after 3.5 years of treatment
10.1172/jci184171 · 2024 · External reference
Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: interim results of a long-term registry-based study
10.1016/j.jcf.2023.03.002 · 2023 · External reference
Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children aged ≥6 years with cystic fibrosis and at least one F508del allele: a phase 3, open-label clinical trial
10.1164/rccm.202301-0021oc · 2023 · External reference
Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST)
10.1016/s2213-2600(14)70218-8 · 2014 · External reference
Effect of elexacaftor/tezacaftor/ivacaftor on annual rate of lung function decline in people with cystic fibrosis
10.1016/j.jcf.2022.12.009 · 2023 · External reference
Unresolved reference
2024 · External reference
Unresolved reference
2024 · External reference
Unresolved reference
2025 · External reference
Unresolved reference
2024 · External reference
Unresolved reference
2025 · External reference
Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children ≥6 years with cystic fibrosis and at least one F508del allele: a 192-week, phase 3, open-label extension study
10.1164/rccm.202502-0512oc · 2025 · External reference
A survey assessing the impact of COVID-19 and elexacaftor/tezacaftor/ifavacaftor on both physical and mental health in adults with cystic fibrosis
10.1002/ppul.26260 · 2023 · External reference
Behavioural and sleep issues after initiation of elexacaftor-tezacaftor-ivacaftor in preschool-age children with cystic fibrosis
10.1016/s0140-6736(24)01134-6 · 2024 · External reference
Effects of elexacaftor/tezacaftor/ivacaftor therapy on mental health of patients with cystic fibrosis
10.3389/fphar.2023.1179208 · 2023 · External reference
Elexacaftor/tezacaftor/ivacaftor treatment and depression-related events
10.1164/rccm.202308-1525oc · 2024 · External reference
Prevalence of depression and anxiety in patients with cystic fibrosis and parent caregivers: results of the International Depression Epidemiological Study across nine countries
10.1136/thoraxjnl-2014-205983 · 2014 · External reference
Mental health changes in adolescents and adults with cystic fibrosis after initiation of elexacaftor/tezacaftor/ivacaftor therapy: insights from the longitudinal resilience impacted by positive stressful events (RISE) study
10.1016/j.chpulm.2025.100146 · 2025 · External reference
Longitudinal effects of elexacaftor/tezacaftor/ivacaftor: multidimensional assessment of neuropsychological side effects and physical and mental health outcomes in adolescents and adults
10.1016/j.chest.2023.10.043 · 2024 · External reference
In vitro modulator responsiveness of 655 CFTR variants found in people with cystic fibrosis
10.1016/j.jcf.2024.02.006 · 2024 · External reference
Theratyping cystic fibrosis patients to guide elexacaftor/tezacaftor/ivacaftor out-of-label prescription
10.1183/13993003.00110-2023 · 2023 · External reference
Elexacaftor/tezacaftor/ivacaftor efficacy in intestinal organoids with rare CFTR variants in comparison to CFTR-F508del and CFTR-wild type controls
10.1016/j.jcf.2024.09.019 · 2025 · External reference
Impact of the expanded label for elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with no F508del variant in the USA
10.1183/13993003.01146-2024 · 2024 · External reference
Elexacaftor/tezacaftor/ivacaftor for cystic fibrosis and rare CFTR variants
10.1093/ajrccm/aamaf001 · 2026 · External reference
The French compassionate programme of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis with advanced lung disease and no F508del CFTR variant
10.1183/13993003.02437-2022 · 2023 · External reference
Unresolved reference
2025 · External reference
Elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis harbouring two CFTR class I variants: real-world data from the French compassionate programme
10.1016/j.eclinm.2025.103476 · 2025 · External reference
In silico analysis and theratyping of an ultra-rare CFTR genotype (W57G/A234D) in primary human rectal and nasal epithelial cells
10.1016/j.isci.2023.108180 · 2023 · External reference
Cystic fibrosis patients of minority race and ethnicity less likely eligible for CFTR modulators based on CFTR genotype
10.1002/ppul.25285 · 2021 · External reference
Molecular structures reveal synergistic rescue of Delta508 CFTR by Trikafta modulators
10.1126/science.ade2216 · 2022 · External reference
The future of cystic fibrosis treatment: from disease mechanisms to novel therapeutic approaches
10.1016/s0140-6736(23)01608-2 · 2023 · External reference
CFTR Cl- channel function in native human colon correlates with the genotype and phenotype in cystic fibrosis
10.1053/j.gastro.2004.s07.006 · 2004 · External reference
Mutations in the cystic fibrosis transmembrane regulator gene and in vivo transepithelial potentials
10.1164/rccm.200509-1377oc · 2006 · External reference
Effect of elexacaftor/tezacaftor/ivacaftor on airway and systemic inflammation in cystic fibrosis
10.1136/thorax-2022-219943 · 2023 · External reference
WS19.01 stabilizers of CFTR NBD1 synergize with galicaftor (SION-2222) or SION-109 to enable full correction of ΔF508-CFTR
10.1016/j.jcf.2025.03.671 · 2025 · External reference
Sweat chloride reflects CFTR function and correlates with clinical outcomes following CFTR modulator treatment
10.1016/j.jcf.2024.12.006 · 2025 · External reference
Progress of personalized medicine of cystic fibrosis in the times of efficient CFTR modulators
10.1186/s40348-025-00194-0 · 2025 · External reference
Personalized CFTR modulator therapy for G85E and N1303K homozygous patients with cystic fibrosis
10.3390/ijms241512365 · 2023 · External reference
Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening
10.1164/rccm.200901-0069oc · 2009 · External reference
Cystic fibrosis
10.1038/s41572-024-00538-6 · 2024 · External reference
Magnetic resonance imaging detects progression of lung disease and impact of newborn screening in preschool children with cystic fibrosis
10.1164/rccm.202102-0278oc · 2021 · External reference
Phase 3 open-label clinical trial of elexacaftor/tezacaftor/ivacaftor in children aged 2-5 years with cystic fibrosis and at least one F508del allele
10.1164/rccm.202301-0084oc · 2023 · External reference
Safety and efficacy of ivacaftor in infants aged 1 to less than 4 months with cystic fibrosis
10.1016/j.jcf.2024.03.012 · 2024 · External reference
Effects of lumacaftor/ivacaftor on cystic fibrosis disease progression in children 2 through 5 years of age homozygous for F508del-CFTR: a phase 2 placebo-controlled clinical trial
10.1513/annalsats.202208-684oc · 2023 · External reference
Long-term impact of lumacaftor/ivacaftor treatment on cystic fibrosis disease progression in children 2 through 5 years of age homozygous for F508del-CFTR: a phase 2, open-label clinical trial
10.1513/annalsats.202402-201oc · 2024 · External reference
Clinical effectiveness of elexacaftor/tezacaftor/ivacaftor in 6 to 11-year-olds with cystic fibrosis
10.1513/annalsats.202501-103oc · 2025 · External reference
In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis
10.1126/scitranslmed.aau7531 · 2019 · External reference
Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs
10.1126/science.1163600 · 2008 · External reference
Perinatal dysfunction of innate immunity in cystic fibrosis
10.1126/scitranslmed.adk9145 · 2025 · External reference
Use of elexacaftor/tezacaftor/ivacaftor combination in pregnancy
10.1007/s00404-023-06962-5 · 2024 · External reference
Maternal and fetal outcomes following elexacaftor-tezacaftor-ivacaftor use during pregnancy and lactation
10.1016/j.jcf.2021.03.006 · 2021 · External reference
First real-world study of fetal therapy with CFTR modulators in cystic fibrosis: report from the MODUL-CF study
10.1016/j.jcf.2025.03.009 · 2025 · External reference
Adapting the cystic fibrosis care model: perspectives from people with CF, caregivers, and members of CF care teams
10.1016/j.jcf.2024.09.002 · 2024 · External reference
Cystic Fibrosis Foundation position paper: redefining the cystic fibrosis care team
10.1016/j.jcf.2024.09.011 · 2024 · External reference
Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials
10.1016/s2213-2600(22)00434-9 · 2023 · External reference
Impact of discontinuing both hypertonic saline and dornase alfa after elexacaftor-tezacaftor-ivacaftor in cystic fibrosis
10.1513/annalsats.202404-366oc · 2024 · External reference
Baseline characteristics and self-reported use of chronic daily therapies of the home reported outcomes with CFTR modulator therapy (HERO-2) cohort
10.1002/ppul.71190 · 2025 · External reference
Effect of elexacaftor-tezacaftor-ivacaftor on body weight and metabolic parameters in adults with cystic fibrosis
10.1016/j.jcf.2021.11.012 · 2022 · External reference
Dietary intake remains unchanged while nutritional status improves in children and adults with cystic fibrosis on elexacaftor/tezacaftor/ivacaftor
10.1016/j.clnu.2025.04.027 · 2025 · External reference
Development of metabolic syndrome in people with cystic fibrosis one year after exposure to elexacaftor-tezacaftor-ivacaftor
10.1016/j.jcf.2024.09.022 · 2025 · External reference
Cardiometabolic risk factors in adults with cystic fibrosis undergoing elexacaftor/tezacaftor/ivacaftor therapy
10.1016/j.jcf.2024.11.009 · 2025 · External reference
Prevalence, risk factors and outcomes of cardiac disease in cystic fibrosis: a multinational retrospective cohort study
10.1183/13993003.00174-2023 · 2023 · External reference
Elevated rates and earlier onset of non-pulmonary comorbidities in adults with cystic fibrosis: a population-based study
10.1513/annalsats.202502-170oc · 2025 · External reference
Glycemia and insulin secretion in cystic fibrosis 2 years after elexacaftor/tezacaftor/ivacaftor: PROMISE-ENDO
10.1210/clinem/dgae857 · 2025 · External reference
The effect of elexacaftor/tezacaftor/ivacaftor (ETI) on glycemia in adults with cystic fibrosis
10.1016/j.jcf.2021.09.001 · 2022 · External reference
Impact of elexacaftor/tezacaftor/ivacaftor on glucose tolerance and abnormal glucose metabolism: a phase 3b, open-label clinical trial
10.1164/rccm.202411-2312oc · 2025 · External reference
Risk factors for chronic kidney disease in adults with cystic fibrosis
10.1164/rccm.201105-0932oc · 2011 · External reference
Consensus document for the selection of lung transplant candidates: an update from the International Society for Heart and Lung Transplantation
10.1016/j.healun.2021.07.005 · 2021 · External reference
Lung transplant referral for individuals with cystic fibrosis: Cystic Fibrosis Foundation consensus guidelines
10.1016/j.jcf.2019.03.002 · 2019 · External reference
Cancer in cystic fibrosis: a narrative review of prevalence, risk factors, screening, and treatment challenges: adult cystic fibrosis series
10.1016/j.chest.2021.09.003 · 2022 · External reference