Skip to content
Log in
Sponsored AI AM CLAUDE, A Beginner's Guide: Learn the basics of working with AI AI AM CLAUDE, A Beginner's Guide: Learn the basics of working with AI
Review

Journal of Cystic Fibrosis

Kevin W. Southern Carlo Castellani E. Lammertyn Alan R. Smyth D. VanDevanter S. van Koningsbruggen-Rietschel J. Barben Amanda Bevan Edwin J. Brokaar Sarah Collins Gary J. Connett T. Daniels Jane Davies D. Declercq S. Gartner A. Gramegna N. Hamilton J. Hauser N. Kashirskaya L. Kessler Jacqueline Lowdon Halyna Makukh Clémence Martin Lisa Morrison D. Nazareth J. A.A. Ciaran O'Neill Bb E. Owen Helen Oxley Dd Karen S. Raraigh Ee C. Ff Karen L. Robinson Jobst Röhmel Carsten Schwarz Isabelle Sermet Jj Michal Shteinberg Kk Ian Sinha Ll Constance Takawira P. van Mourik M. Verkleij Michael D. Waller Oo Alistair Duff
· 0 citations · 133 references

TL;DR

Interim standards to guide healthcare professionals in the provision of variant-specific therapy for people with CF are produced, established using evidence from systematic reviews and expert opinion.

Semantic Scholar
View source

Similar papers

Review Sep 2026

Comment on “Translating Emerging Data for Fetal Treatment of Cystic Fibrosis”

Prenatal CFTRm therapy is promising but remains investigational, and care should include multidisciplinary counseling, genetic confirmation of fetal CF, maternal liver function monitoring, fetal ultrasound surveillance, and postnatal follow-up, according to standardized guidelines.

M. E. Norton · 0 citations
Open access Aug 2026

Triple CFTR modulator therapy in severe cystic fibrosis-associated liver disease: a case report of an adverse outcome and the role of adherence to follow-up

Background. Cystic fibrosis (CF) is a multisystem disease in which liver involvement occurs in 20-40% of patients. The introduction of CFTR modulator therapy has substantially improved the prognosis of patients with CF; however, patients with established cirrhosis have typically been excluded from clinical trials, whic...

Z. Merzhoeva, O. Suvorova, I. A. Levina et al. · 0 citations
Open access Sep 2026

Bridging the Gap in Eastern European Cystic Fibrosis Care: How Newborn Screening and Advanced CFTR Modulation Shape the Clinical Landscape in Western Romania

Cystic fibrosis characterization in western Romania demonstrates rapid modulator uptake and preserved lung function while highlighting persistent gaps in nutritional status, microbiological burden, and case detection relative to Western European populations.

C. Marinău, C. Sava, A. Iuhas et al. · 0 citations
Open access Sep 2026

The effectiveness of a generic CFTR-modulator in a prolonged multicenter study

Introduction. Prior to recent years, treatment of cystic fibrosis (CF) consisted primarily of symptom management. The pathogenetic therapy with CFTR modulators has emerged as a revolutionary method, supplying the capacity to correct the underlying CFTR chloride channel dysfunction. Aim. To evaluate the effic...

E. Kondratyeva, A. Voronkova, E. Zhekayte et al. · 0 citations
Open access Oct 2026

Challenges in targeted therapy for cystic fibrosis in a patient with the complex allele [L467F; F508del]: from failure of first-line modulator therapy to successful use of vanzacaftor+tezacaftor+deutivacaftor combination

Cystic fibrosis (CF) is the most common inherited autosomal recessive disorder, caused by mutations in the CFTR gene. The introduction of CFTR modulators has fundamentally changed the prognosis and quality of life of patients. However, the presence of complex alleles may significantly affect the efficacy of targeted th...

А. V. Nistarova, S. I. Petrova, Y. V. Peshekhonova et al. · 0 citations

We use cookies to run the site and, with your consent, for analytics and to show ads. See our Cookie Policy.