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The United Nations convention on rare diseases—A framework for research prioritization

Citation: Junaid M, Downs J, Groza T, Lassmann T, Baker S, et al. The United Nations convention on rare diseases—A framework for research

Cardiac monitoring safety assessment framework for early phase group a streptococcal vaccine trials

This paper presents a comprehensive cardiac safety framework for early clinical development of Streptococcus pyogenes (Group A Streptococcus) vaccines, endorsed by the Strep A Vaccine Global Consortium and the Australian Strep A Vaccine Initiative. Given historical concerns about vaccine-associated acute rheumatic fever, we have established standardized echocardiography protocols integrated with clinical assessment for monitoring cardiac safety in early-phase vaccine trials.

Resilience and mental health among care leavers: Role of social inclusion, self-determination, and independent living skills

Young people transitioning from out-of-home care (OHC) frequently experience poor mental health and resilience due to adverse childhood experiences (ACEs). However, there is limited understanding of the factors that mediate and moderate these outcomes. This is the first study to integrate linked administrative and longitudinal data to examine the mediation and moderation effects of placement stability, independent living skills (ILS), social inclusion, and self-determination when examining the association between ACEs and care status on mental health and resilience.

Prioritizing high-risk populations for soil-transmitted helminth control in the Western Pacific Region

To achieve targets set within the 2030 Sustainable Development Agenda and the 2021–2030 Neglected Tropical Diseases (NTD) Roadmap, the World Health Organization identify the need for improved data analytics to inform NTD control programs. 

Cochlear implantation in children under 12 months of age: surgical outcomes and considerations—a Western Australian perspective

The introduction of universal newborn hearing screening has allowed for early identification and diagnosis of children with severe-profound hearing loss. This study aims to provide the first Western Australian perspective on the surgical feasibility and safety of cochlear implantation before 12 months of age.

Parenting in the age of social media: The buffering effect of parental self-efficacy on the relationship between parental social media use and parent child-relationship quality

The widespread use of technology in daily life has raised concerns about its potential to disrupt social relationships, particularly within one of the most important human relationships: the parent-child relationship. This study assesses whether parental social media use (measured by a novel parental social media intensity scale) affects the parent-child relationship (measured by the child-parent relationship scale - short form), and whether parental self-efficacy (PSE, measured by the parenting sense of competence scale) moderates this effect.

Heterogeneity and distribution characteristics of tertiary lymphoid structures predict prognostic outcome in esophageal squamous cell carcinoma

Tertiary Lymphoid Structures (TLSs) are ectopic lymphoid aggregates that form within the tumor microenvironment (TME) and are increasingly recognized as potential prognostic biomarkers in various cancers. However, the spatial heterogeneity and prognostic value of TLSs in esophageal squamous cell carcinoma (ESCC) remain poorly defined. This study aimed to characterize the spatial distribution patterns of TLSs and tumor-infiltrating lymphocytes (TILs), and to establish a refined prognostic model for ESCC patients in both surgery-only and neoadjuvant therapy cohorts.

Sex assigned at birth may modify health-related quality of life in children treated with peanut oral immunotherapy

The high burden of peanut allergy underscores the need for treatment options that improve patient health-related quality of life (HRQL). However, the modifying effect of sex assigned at birth on treatment-related outcomes remains poorly understood. We sought to investigate whether sex modifies treatment effect on the change in overall and subdomain HRQL during the PPOIT-003 trial.

Climate change policies fail to protect child health

National policies are essential for countries to adapt to the negative health impacts of climate change. Children are disproportionately affected by these impacts and must be at the heart of adaptation policies to address their vulnerabilities. Adaptation commitments worldwide are integrated into national adaptation plans, nationally determined contributions, national communications, and other multisectoral policies. We aimed to evaluate how effectively national climate change policies worldwide plan to protect child health, considering a range of determinants for successful child-health adaptation.

Longitudinal Profiling of the Human Milk Microbiome from Birth to 12 Months Reveals Overall Stability and Selective Taxa-Level Variation

Human milk bacteria contribute to gut microbiome establishment in breastfed infants. Although breastfeeding is recommended throughout infancy, temporal variation in the milk microbiome-particularly beyond solid food introduction-remains understudied. We analyzed 539 milk samples from 83 mother-infant dyads between 1 week and 12 months postpartum using full-length 16S rRNA gene sequencing.