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Beyond Seizures as an Outcome Measure: A Global Severity Scoring System for CDKL5 Deficiency Disorder

CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy (DEE) associated with multiple impairments and comorbidities. Outcome measures for disease-modifying clinical trials for DEEs should measurably capture a spectrum of caregiver priorities and be externally validated.

Mapping Bacillus Calmette-Guérin vaccination coverage in Africa from 1990 to 2022: a novel spatiotemporal modelling study

Bacillus Calmette-Guérin (BCG) protects children from severe tuberculosis and remains the only licensed vaccine for tuberculosis. Subnational estimates of BCG coverage are essential for identifying underserved populations across Africa. This study aimed to map BCG vaccination coverage in Africa from 1990 to 2022. 

A meta-analytic review and conceptual model of the antecedents and outcomes of goal adjustment in response to striving difficulties

There is growing interest in how and why individuals adjust their goals in response to difficulties encountered during goal striving and the outcomes of such adjustments; however, research on these topics is fragmented across theoretical perspectives and life domains. 

Hospitalizations Following Complex Hip Surgery in Children with Intellectual Disability: A Self-Controlled Case Series Analysis

To evaluate the associations between complex hip surgery and subsequent hospitalizations in children with intellectual disability, including a subset of children with cerebral palsy.

Perturbation of the T Cell Compartment Underlies Rheumatic Fever Pathogenesis

This prospective cohort study, which recruited participants with definite ARF in Australia and Aotearoa New Zealand, profiled circulating immune molecules and cells to inform disease mechanisms and future druggable pathways.

Antimicrobial Resistance in Enterobacterales, Acinetobacter spp. and Pseudomonas aeruginosa Isolates From Bloodstream Infections in Australian Children, 2013–2021

Gram-negative bloodstream infections are associated with significant morbidity and mortality in children. Increasing antimicrobial resistance (AMR) is reported globally, yet efforts to track pediatric AMR at a national level over time are lacking.  

Antimicrobial Resistance in Enterobacterales, Acinetobacter spp. and Pseudomonas aeruginosa Isolates From Bloodstream Infections in Australian Children, 2013–2021

Gram-negative bloodstream infections are associated with significant morbidity and mortality in children. Increasing antimicrobial resistance (AMR) is reported globally, yet efforts to track pediatric AMR at a national level over time are lacking.

Coupling of response biomarkers between tumor and peripheral blood in patients undergoing chemoimmunotherapy

Platinum-based chemotherapy in combination with anti-PD-L1 antibodies has shown promising results in mesothelioma. However, the immunological mechanisms underlying its efficacy are not well understood and there are no predictive biomarkers to guide treatment decisions.

The Feasibility of Personalized Endpoints in Assessing Treatment Outcomes for Rare Diseases: A Pilot Study of Goal Attainment Scaling in SCN2A-Associated

For individuals living with rare neurodevelopmental disorders, particularly those who are at the most severe end of the spectrum, standardized outcome measures may lack the sensitivity to capture small but meaningful changes. 

Prevalence of dementia among Indigenous populations of countries with a very high Human Development Index: a systematic review

Dementia is a health priority for Indigenous peoples. Here, we reviewed studies on the prevalence of dementia or cognitive impairment among Indigenous populations from countries with a very high Human Development Index (≥0·8). Quality was assessed using the Joanna Briggs Institute risk-of-bias tool and CONSolIDated critERia for strengthening the reporting of health research involving Indigenous peoples (CONSIDER), with oversight provided by an Indigenous Advisory Board.