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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

Rare Occurrence of Congenital Neuroblastoma and Tuberous Sclerosis

Citation: Gardner M, Shah S, Jain N, Bynevelt M. Rare Occurrence of Congenital Neuroblastoma and Tuberous Sclerosis. Pediatr Neurol. 2026;176:62-3.

Move to improve: protocol for a single-arm, pragmatic feasibility trial of an individualised physical activity programme for children with chronic conditions

Physical activity improves physical and psychosocial outcomes in healthy children and in children with a range of chronic health conditions. Unfortunately, children with chronic health conditions have lower levels of physical activity compared to their healthy peers due to multiple restrictions in physical activities and therefore tend to have lower levels of physical activity compared with their peers. This paper describes the protocol for Move to Improve, a pragmatic trial of an individualised physical activity intervention for children with chronic health conditions.

A Scoping Review of Digital Interventions Targeting Anxiety in Young Children

Anxiety is a leading mental health concern in childhood. Whilst a range of therapeutic approaches effectively reduce anxiety in young children, several barriers impact their implementation into practice. Digital interventions could help overcome some of these challenges; however, whether these can effectively target anxiety for children with and without Neurodevelopmental Conditions is unknown.

A small molecule inhibitor of RNA-binding protein IGF2BP3 shows anti-leukemic activity

The RNA-binding protein IGF2BP3 is an oncofetal protein overexpressed in B-acute lymphoblastic leukemia and is critical for leukemogenesis in experimental models. With cancerspecific expression, functional dispensability for normal development, and an unleveraged prooncogenic function in mRNA homeostasis, IGF2BP3 represents an excellent target.

Core Outcome Sets for Pediatric Perioperative Research: An International Stakeholder Engagement Exercise and Consensus Report from the Pediatric Perioperative Outcomes Group

dentifying the outcomes that matter in clinical research is important, especially those that matter to patients and their parents/guardians. Consistency in outcome reporting enables meaningful assessments of interventions and facilitates comparison of results across trials. The aim of this study was to develop core outcome sets for pediatric perioperative research.

Twenty-One Years and Still Going Strong: A Qualitative Study Exploring the Contribution of Young Adult, Adolescent, and Stakeholder Involvement to the Resilience

Adolescence is a period of rapid transformation when meeting targets for optimal diabetes care is often challenging due to competing life demands. For more than two decades a diabetes transition clinic in Sydney, Australia, has sustained positive outcomes and demonstrated aspects of resilience in the care of individuals living with type 1 diabetes (T1D) who have transitioned from paediatric to adult care. Many studies have focused on resilience in acute care setting showever, studies that examine the factors that support resilience in settings that care for individuals with long-term, chronic conditions such as T1D are lacking.

Understanding the Determinants of Use of the Australian Guide to Diagnosis of Fetal Alcohol Spectrum Disorder: Informing the Review, Development and Dissemination

The Australian Guide to the Diagnosis of fetal alcohol spectrum disorder (FASD), developed in 2016, is currently under review. This study aimed to understand how the Guide is used in practice and identify factors influencing its implementation.

Enhancing Anesthesia Research: The Imperative of Consumer Engagement Into Clinical Research

Britta Regli-von Ungern-Sternberg AM FAHMS MD, PhD, DEAA, FANZA Chair of Paediatric anaesthesia, University of Western Australia; Consultant

Disordered eating in autistic trans and gender diverse people: a lived experience-led scoping review

This lived experience-led scoping review explores the evidence base related to eating disorders/disordered eating behaviours in Autistic trans and gender diverse (TGD) people. This review highlights the currently available data on eating disorder prevalence rates, comparisons with allistic and cisgender groups, drivers and maintenance factors, the relationship between eating disorders and gender-affirming medical care, and treatment outcomes in this population.