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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:
Background: In phase 2 trials in people with cystic fibrosis aged 18 years and older, vanzacaftor-tezacaftor-deutivacaftor has been shown to be a safe and effective, once-daily cystic fibrosis transmembrane conductance regulator (CFTR) modulator. Restoring normal CFTR function early in life has the potential to prevent manifestations of cystic fibrosis. We aimed to evaluate the safety, tolerability, efficacy, and pharmacokinetics of vanzacaftor-tezacaftor-deutivacaftor in children with cystic fibrosis aged 6-11 years.
View Article and Find Full Text PDFVanzacaftor-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 PDFA FET-based flexible biosensor system for dynamic behavior observation of lipid membrane with nanoparticles .
Lab Chip
January 2025
State Key Laboratory of Precision Measuring Technology and Instruments, School of Precision Instruments and Optoelectronics Engineering, Tianjin University, Tianjin 300072, China.
Nanoparticles have become widely used materials in various fields, yet their mechanism of action at the cellular level after entering the human body remains unclear. Accurately observing the effect of nanosize dimensions on particle internalization and toxicity in cells is crucial, particularly under the conditions of biological activity. With the aim of helping to study the interactions between nanoparticles of varying sizes and active cell membranes, we propose a flexible biosensor system based on a field effect transistor (FET).
View Article and Find Full Text PDFCognitive Impairment in MASLD is associated with Amygdala-Related Connectivity Dysfunction in the Prefrontal and Sensory Cortex.
J Integr Neurosci
December 2024
Department of Radiology, The Affiliated Hospital of Hangzhou Normal University, 310015 Hangzhou, Zhejiang, China.
Background: Metabolic dysfunction-associated steatotic liver disease (MASLD) is a common metabolism-related multisystem clinical disorder, often accompanied by a high comorbidity of mild cognitive impairment (MCI). Increasing evidence suggests that the amygdala is crucial in cognitive processing during metabolic dysfunction. Nevertheless, the role of the amygdala in the neural mechanisms of MASLD with MCI (MCI_MASLD) remains unclear.
View Article and Find Full Text PDFInternal Ophthalmoplegic Migraine During Pregnancy: A Clinical Case.
Neurol Int
December 2024
Universidad Simón Bolívar, Facultad de Ciencias de la Salud, Barranquilla 080001, Atlántico, Colombia.
Background: Ophthalmoplegic migraine (OM) is an uncommon variant of migraine characterised by headache and cranial nerve palsy, posing significant diagnostic and therapeutic challenges.
Objective: This study aimed to describe an extremely rare OM variant with a partial therapeutic response.
Clinical Case: A 34-year-old pregnant woman in gestational week 19.
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