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Initial Validation and Reliability of the CDKL5 Deficiency Disorder Hand Function Scale (CDD-Hand)

Pathogenic variants in the CDKL5 gene result in CDKL5 deficiency disorder (CDD), which is characterized by early-onset epilepsy, severe developmental delay, and often, cortical visual impairment. Validated clinical outcome measures are needed for future clinical trials to be successful. This study aimed to adapt the Rett Syndrome Hand Function Scale for CDKL5 deficiency disorder and evaluate its feasibility, acceptability, content validity, and reliability.

High prevalence of hearing loss in urban Aboriginal infants: the Djaalinj Waakinj cohort study

Chris Deborah Tamara Brennan-Jones Lehmann Veselinovic PhD AO, MBBS, MSc BSc(Hons) MClinAud PhD Head, Ear and Hearing Health Honorary Emeritus Fellow

Urinary albumin/creatinine ratio tertiles predict risk of diabetic retinopathy progression: a natural history study from the Adolescent Cardio-Renal Intervention Trial (AdDIT) observational cohort

We hypothesised that adolescents with type 1 diabetes with a urinary albumin/creatinine ratio (ACR) in the upper tertile of the normal range (high ACR) are at greater risk of three-step diabetic retinopathy progression (3DR) independent of glycaemic control.

Association between early respiratory viral infections and structural lung disease in infants with cystic fibrosis

Infants with cystic fibrosis (CF) develop structural lung disease early in life, and viral infections are associated with progressive lung disease. We hypothesized that the presence of respiratory viruses would be associated with structural lung disease on computed tomography (CT) of the chest in infants with CF.

Reducing exacerbations in children and adults with primary ciliary dyskinesia using erdosteine and/or azithromycin therapy (REPEAT trial): study protocol for a multicentre, double-blind, double-dummy, 2×2 partial factorial, randomised controlled trial

Primary ciliary dyskinesia (PCD) is a rare, progressive, inherited ciliopathic disorder, which is incurable and frequently complicated by the development of bronchiectasis. There are few randomised controlled trials (RCTs) involving children and adults with PCD and thus evidence of efficacy for interventions are usually extrapolated from people with cystic fibrosis.

International comparison of glycaemic control in people with type 1 diabetes: an update and extension

o update and extend a previous cross-sectional international comparison of glycaemic control in people with type 1 diabetes. Methods: Data were obtained for 520,392 children and adults with type 1 diabetes from 17 population and five clinic-based data sources in countries or regions between 2016 and 2020.

Variation in the prevalence of different forms of bullying victimisation among adolescents and their associations with family, peer and school connectedness: a population-based study in 40 lower and middle income to high-income countries (LMIC-HICs)

This study aimed to estimate the prevalence of different forms of bullying victimization experiences and their association with family functioning, peer relationships and school connectedness among adolescents across 40 lower and middle income to high-income countries (LMIC-HICs).

Looking beyond: complex holistic care needs of Syrian and Iraqi refugee children and adolescents

Protracted international conflict has seen escalating numbers of displaced and resettled Syrian and Iraqi refugees, raising concerns for their health and well-being. This paper describes the demographic and clinical profiles of recently resettled Syrian and Iraqi refugee children and adolescents across physical, psychosocial, developmental and educational domains using standardised multidisciplinary assessments.

Assessing the utility of routine viral surveillance performed in children undergoing autologous stem cell transplantation at a single centre

We assessed the utility of routine viral surveillance for cytomegalovirus, Epstein–Barr virus and human adenovirus in children <16 years, undergoing autologous stem cell transplantation (ASCT) at a single centre over a 10-year period. A total of 85 ASCT were performed in 65 patients.

Evidence of maternal transfer of antigen-specific antibodies in serum and breast milk to infants at high-risk of S. pneumoniae and H. influenzae disease

Children in low-mid income countries, and First Nations children in high-income countries, experience disproportionately high rates of Streptococcus pneumoniae and Haemophilus influenzae infections and diseases including pneumonia and otitis media.