Real-world disparities and ethical considerations with access to CFTR modulator drugs: mind the gap!

M Zampoli, BM Morrow, G Paul - Frontiers in Pharmacology, 2023 - frontiersin.org
The third Sustainable Development Goal (SDG), to ensure healthy lives and promote well-
being for all at all ages, has particular relevance and implementation challenges amongst …

Efficacy and safety of elexacaftor-tezacaftor-ivacaftor in the treatment of cystic fibrosis: a systematic review

N Kapouni, M Moustaki, K Douros, I Loukou - Children, 2023 - mdpi.com
Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) is a new CFTR (Cystic Fibrosis
Transmembrane Conductance Regulator) modulator treatment, used over the last few years …

Prevalence, risk factors and outcomes of cardiac disease in cystic fibrosis: a multinational retrospective cohort study

F Frost, D Nazareth, L Fauchier, D Wat… - European …, 2023 - publications.ersnet.org
Background Although people living with cystic fibrosis (PwCF) often have some risk factors
for cardiovascular disease, including diabetes and chronic inflammation, little is known …

[HTML][HTML] Elexacaftor/Tezacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for the F508del Mutation and Advanced Lung Disease: A 48-Week …

V Carnovale, P Iacotucci, V Terlizzi… - Journal of clinical …, 2022 - mdpi.com
Background: Elexacaftor/tezacaftor/ivacaftor (ETI) is the newest cystic fibrosis
transmembrane conductance regulator (CFTR) modulator drug approved for the treatment of …

Lung function decline in cystic fibrosis: impact of data availability and modeling strategies on clinical interpretations

R Szczesniak, ER Andrinopoulou, W Su… - Annals of the …, 2023 - atsjournals.org
Rationale Studies estimating the rate of lung function decline in cystic fibrosis have been
inconsistent regarding the methods used. How the methodology used impacts the validity of …

Impact of airway inflammation on the efficacy of CFTR modulators

CMP Ribeiro, M Gentzsch - Cells, 2021 - mdpi.com
Defective CFTR biogenesis and activity in cystic fibrosis airways leads to airway dehydration
and impaired mucociliary clearance, resulting in chronic airway infection and inflammation …

Tezacaftor/Ivacaftor therapy has negligible effects on the cystic fibrosis gut microbiome

R Marsh, C Dos Santos, L Hanson, C Ng… - Microbiology …, 2023 - journals.asm.org
People with cystic fibrosis (pwCF) experience a range of persistent gastrointestinal
symptoms throughout life. There is evidence indicating interaction between the microbiota …

Diagnosis and management of cystic fibrosis exacerbations

T Milinic, OJ McElvaney… - Seminars in respiratory and …, 2023 - thieme-connect.com
With the improving survival of cystic fibrosis (CF) patients and the advent of highly effective
cystic fibrosis transmembrane conductance regulator (CFTR) therapy, the clinical spectrum …

Therapeutic interventions alter ecological interactions among cystic fibrosis airway microbiota

PM Ho, RR Nazeer, M Welch - Frontiers in Microbiology, 2023 - frontiersin.org
The airways of people with cystic fibrosis (CF) often harbor a diverse microbiota and in
recent years, much effort has been invested in cataloguing these. In spite of providing a …

Proteomics profiling of inflammatory responses to elexacaftor/tezacaftor/ivacaftor in cystic fibrosis

H Ozuna, D Bojja, S Partida-Sanchez… - Frontiers in …, 2025 - frontiersin.org
Background CFTR modulator therapies have positive clinical outcomes, yet chronic
inflammation and bacterial infections persist in people with CF (pwCF). How elexacaftor …