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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.
This study aimed to develop a consensus-derived minimum data set for childhood stroke and establish an agreed set of tools to measure key functional outcomes identified as important by childhood stroke survivors and their families.
People with intellectual disability experience significant gaps in healthcare delivery resulting in poor health outcomes. Appropriately designed healthcare is required to meet the needs of this population and achieve better health outcomes. Little is known about the structure of healthcare delivery for people with intellectual disability and whether it is effective or cost-effective.
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.
This Review describes the burden of cardiovascular disease across Indigenous populations and contextualizes the potential drivers and contributors to inequalities between Indigenous and non-Indigenous populations. The focus is on Indigenous populations across Aotearoa New Zealand, Australia, Canada and the USA, including Aboriginal and Torres Strait Islander, American Indian, Alaska Native, Canadian First Nations, Inuit, Métis, Māori, Native Hawaiian and Pacific Islander peoples.
Pneumococcal conjugate vaccine (PCV) prevents pneumococcal disease and pneumonia, but indirect effects are poorly understood in low-coverage, high-burden settings like Papua New Guinea (PNG). PNG introduced 13-valent PCV (PCV13) in 2014. We aimed to assess direct and indirect effectiveness of PCV13 against vaccine-type pneumococcal carriage among children with pneumonia or suspected meningitis in PNG
Although evidence supports clinicians to "safely do less" for febrile infants assessed as low risk of serious bacterial infection (SBI), early discharge may increase caregiver concern and reduce satisfaction with care. We captured the self-reported satisfaction and concerns for families enrolled in the study of fever, blood cultures and readiness for discharge in infants less than 3 months old (FeBRILe3), a prospective safety assessment of early discharge of low-risk febrile infants, to aid evaluation of this practice.
To summarize recent evidence in pediatric total intravenous anesthesia (TIVA), highlighting advances in pharmacokinetics-pharmacodynamics, target-controlled infusion (TCI), electroencephalography (EEG)-guided titration, emerging agents, safety, and sustainability, and to provide clinicians with an updated, practical framework for pediatric TIVA practice.
Leadership in paediatric anaesthesia is undergoing rapid transformation as clinical complexity, workforce expectations, and organizational structures evolve. This review synthesizes recent developments and highlights the competencies required for effective leadership in this high-stakes specialty.
To better characterise prematurity-associated lung disease, adult spirometry phenotype classifications (obstructive lung disease, preserved ratio impaired spirometry and dysanapsis) have been applied to children born preterm. It is unknown how these phenotypes track over time.