Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Profiling the response to lumacaftor-ivacaftor in children with cystic between fibrosis and new insight from a French-Italian real-life cohort.

Pediatric pulmonology - 1 Dec 2022

Cornet Matthieu, Robin Geneviève, Ciciriello Fabiana, Bihouee Tiphaine, Marguet Christophe, Roy Valérie, Lebourgeois Muriel, Chedevergne Frédérique, Bonnel Anne Sophie, Kelly Mairead, Reix Philippe, Lucidi Vincenzina, Stoven Véronique, Sermet-Gaudelus Isabelle

Abstract excerpt

INTRODUCTION: Clinical trials for CFTR modulators consider mean changes of clinical status at the cohort level, and thus fail to assess the heterogeneity of the response. We aimed to study the different response profiles to lumacaftor-ivacaftor according to age in children with cystic fibrosis (CF). METHODS: A mathematical framework, including principal component analysis, data clustering, and data completion,...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Aminophenols
Share to Aminopyridines

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Benzodioxoles

Share to Cystic Fibrosis

Share to Cystic Fibrosis Transmembrane Conductance Regulator

Share to Drug Combinations

Share to Fibrosis

Share to Forced Expiratory Volume

Source

Open in PubMed Open full text

Identifiers

PMID: 35996214
DOI: 10.1002/ppul.26123
PMCID: PMC9826158

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Profiling the response to lumacaftor-ivacaftor in children with cystic between fibrosis and new insight from a French-Italian real-life cohort.PMID 35996214Read ↗

Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study.Semantic proximity Read ↗

Real-world data on the efficacy and safety of tezacaftor-ivacaftor in adults living with cystic fibrosis homozygous for F508del and heterozygous for F508del and a residual function mutation.Semantic proximity Read ↗

Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor.Semantic proximity Read ↗

Contributors to the heterogeneity of response to CFTR modulators.Semantic proximity Read ↗

The clinical impact of Lumacaftor-Ivacaftor on structural lung disease and lung function in children aged 6-11 with Cystic Fibrosis in a real-world settingSemantic proximity Read ↗

Clinical and functional course of patients with cystic fibrosis treated with lumacaftor/ivacaftor at a children's hospital. A case series.Semantic proximity Read ↗

The clinical impact of Lumacaftor-Ivacaftor on structural lung disease and lung function in children aged 6-11 with cystic fibrosis in a real-world setting.Semantic proximity Read ↗

Pediatric population with cystic fibrosis in the centre of Portugal: candidates for new therapies.Semantic proximity Read ↗

A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR.Semantic proximity Read ↗

Genotype-dependent biochemical response to elexacaftor/tezacaftor/ivacaftor in children with cystic fibrosis: A 24-month real-world study.Semantic proximity Read ↗

Treatment of Cystic Fibrosis Patients Homozygous for F508del with Lumacaftor-Ivacaftor (Orkambi®) Restores Defective CFTR Channel Function in Circulating Mononuclear Cells.Semantic proximity Read ↗

Elexacaftor/tezacaftor/ivacaftor in children aged 6–11 years with cystic fibrosis, at least one F508DEL allele, and advanced lung disease: A 24‐week observational studySemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.