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Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children ≥2 years with cystic fibrosis: 96-week interim results from a phase 3 open-label extension study

Elexacaftor/tezacaftor/ivacaftor(ELX/TEZ/IVA) was safe and efficacious in children with CF 2-5y with at least one F508del allele in 24-week phase 3 Trial 445-111. Children who completed 445-111 were eligible for the long-term safety and efficacy extension trial.

Respiratory disease in cerebral palsy: the overlooked impact of neonatal lung disease

Respiratory disease is a leading cause of hospitalisations in children with cerebral palsy (CP). Over 40% of individuals with CP are born preterm; however, the relationship between prematurity, CP and respiratory disease is unknown.

ORIGIN-1 trial: study protocol for an organoid-guided N-of-1 trial evaluating CFTR modulator response in cystic fibrosis with rare non-F508del mutations

Cystic fibrosis (CF) is a genetic condition caused by mutation of the cystic fibrosis transmembrane regulator (CFTR) gene. Recently licensed modulator therapies target the defective CFTR protein and have transformed the formerly life-limiting trajectory of people with CF (pwCF).

Chlorinated drinking water exposure enriches antimicrobial resistance pathways in the infant gut microbiome: a randomized trial

Water chlorination is essential for controlling harmful microbes in drinking water; however, the antimicrobial effects of chlorine-based disinfectants present in tap water may influence early-life gut microbial ecology.

Animal-free peptones support bacteriophage propagation for therapeutic use without altering bactericidal activity or genomic integrity

Bacteriophage (phage) propagation has traditionally relied on bacterial culture media containing animal-derived ingredients; however, safety concerns with animal-derived materials for production of phages for therapeutic use limit their acceptability. We compared animal-free and traditional media formulations, and evaluated effects on phage yield, bactericidal activity, and genomic characteristics, hypothesizing no significant differences would be observed.