Reports and Findings
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.
Streptococcus pyogenes (Group A Streptococcus, Strep A) is a Gram-positive bacterium and one of the most significant global bacterial pathogens, with disproportionate disease burden affecting low and middle-income countries. Despite substantial mortality and immune sequelae caused by Strep A, no licensed vaccines exist, and correlates of protection remain unestablished.
Acute lymphoblastic leukaemia (ALL) is one of the most treatable forms of paediatric cancer; however, there is a substantial burden of treatment-related toxicities (TRTs). In addition, the long-term changes in children's health-related quality of life (HRQoL) due to toxic treatments are not well understood.
There is a paucity of longitudinal research comparing early-life mental health in rural and metropolitan communities. Living in rural areas is associated with limited access to specialist care, as well as additional stresses such as natural disasters. To address health disparities for rural communities it is critical to understand variance in predictors and outcomes for child mental health.
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.
Craniospinal irradiation (CSI) is a cornerstone of pediatric brain cancer therapy, yet detailed, reproducible protocols for accurate CSI delivery in preclinical mouse models remain scarce, hindering translational research and the development of radiosensitizing strategies. We aimed to establish a clinically relevant, easily replicable radiation therapy protocol for medulloblastoma mouse models, providing a robust platform for preclinical evaluation of novel therapeutic combinations.
A retrospective cohort study was conducted using clinical data from patients attending TB clinics at four public hospitals in Northwest Ethiopia. We modelled BMI trajectories after TB treatment initiation using linear mixed models, with adjustments for potential confounders.
There are scant and variable data concerning the incidence of malaria in infants in endemic areas. This study evaluated the impact of maternal antimalarial treatment given postpartum on malaria incidence and growth/development in infants from coastal Papua New Guinea (PNG).
Children with serious burns often suffer psychosocial consequences. This study evaluated the pilot of a 6-session psychoeducational intervention newly designed to support the wellbeing of children who experienced burns.
The impact of smallpox (variola) on Aboriginal communities in Australia beginning in 1789 was catastrophic and continues to cause intergenerational trauma. Historically biased perspectives and contemporary misinformation of the disease's introduction and spread impede modern-day truth-telling and efforts towards reconciliation and national healing.