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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.
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.
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.
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.
Britta Regli-von Ungern-Sternberg AM FAHMS MD, PhD, DEAA, FANZA Chair of Paediatric anaesthesia, University of Western Australia; Consultant
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.
Dr Jackie Davis BSc (Hons), PGradDipHlthProm, PhD Co-Director, ORIGINS 0478173989 jackie.davis@thekids.org.au Co-Director, ORIGINS Dr Jackie Davis
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.
Convalscent plasma (CP) was identified as a potential therapy for COVID-19 available early in the pandemic.
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.