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Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial.
Lancet Respir Med
December 2024
Population Policy and Practice Department, UCL Great Ormond Street Institute of Child Health, University College London, London, UK; Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK. Electronic address:
Article Synopsis
- Vanzacaftor-tezacaftor-deutivacaftor is a new CFTR modulator showing safety and effectiveness in phase 2 trials for adults with cystic fibrosis, leading to a study evaluating its use in children aged 6-11.
- This phase 3 trial, called RIDGELINE, involved participants from 33 clinical sites across eight countries, focusing on children with specific CFTR variants and stable health conditions.
- The study aimed to assess the drug's safety, tolerability, and efficacy over 24 weeks, with primary outcomes evaluated through various health metrics and participant feedback.
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.
Lancet Respir Med
December 2024
Division of Infection, Immunity and Respiratory Medicine, University of Manchester, Manchester, UK. Electronic address:
Background: The goal of cystic fibrosis transmembrane conductance regulator (CFTR) modulators is to reach normal CFTR function in people with cystic fibrosis. Vanzacaftor-tezacaftor-deutivacaftor restored CFTR function in vitro and in phase 2 trials in participants aged 18 years and older resulting in improvements in CFTR function, as measured by sweat chloride concentrations and lung function as measured by spirometry. We aimed to evaluate the efficacy and safety of vanzacaftor-tezacaftor-deutivacaftor compared with standard of care elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older.
View Article and Find Full Text PDFPsychometric performance of the CFQ-R-8D compared to the EQ-5D-3L and SF-6D in people with cystic fibrosis.
J Patient Rep Outcomes
February 2024
Joe DiMaggio Cystic Fibrosis, Pulmonary and Sleep Center, Hollywood, FL, USA.
Objective: This study aimed to compare the psychometric performance of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions (CFQ-R-8D), a new, condition-specific, preference-based measure, with that of generic preference-based measures EQ-5D-3L and Short Form 6 dimensions (SF-6D).
Methods: Data from three trials of participants with CF aged ≥ 14 years who completed the CFQ-R and EQ-5D-3L or SF-6D were used. Analyses were undertaken to evaluate convergent validity based on correlations with CFQ-R domain scores.
Correlating Cystic Fibrosis Transmembrane Conductance Regulator Function with Clinical Features to Inform Precision Treatment of Cystic Fibrosis.
Am J Respir Crit Care Med
May 2019
1 McKusick-Nathans Institute of Genetic Medicine, School of Medicine.
The advent of precision treatment for cystic fibrosis using small-molecule therapeutics has created a need to estimate potential clinical improvements attributable to increases in cystic fibrosis transmembrane conductance regulator (CFTR) function. To derive CFTR function of a variety of genotypes and correlate with key clinical features (sweat chloride concentration, pancreatic exocrine status, and lung function) to develop benchmarks for assessing response to CFTR modulators. CFTR function assigned to 226 unique genotypes was correlated with the clinical data of 54,671 individuals enrolled in the Clinical and Functional Translation of CFTR (CFTR2) project.
View Article and Find Full Text PDFDecreased mRNA and protein stability of W1282X limits response to modulator therapy.
J Cyst Fibros
September 2019
McKusick-Nathans Institute of Genetic Medicine, Johns Hopkins University School of Medicine, Baltimore, MD, United States. Electronic address:
Background: Cell-based studies have shown that W1282X generates a truncated protein that can be functionally augmented by modulators. However, modulator treatment of primary cells from individuals who carry two copies of W1282X generates no functional CFTR. To understand the lack of response to modulators, we investigated the effect of W1282X on CFTR RNA transcript levels.
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