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ADR3, a next generation i-body to human RANKL, inhibits osteoclast formation and bone resorptionOsteoporosis is a chronic skeletal condition characterized by low bone mass and deteriorated microarchitecture of bone tissue and puts tens of millions of people at high risk of fractures. New therapeutic agents like i-bodies, a class of next-generation single-domain antibodies, are needed to overcome some limitations of conventional treatments.
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Australian children living with rare diseases: health service use and barriers to accessing careChildren with rare diseases experience challenges at home and school and frequently require multi-disciplinary healthcare. We aimed to determine health service utilization by Australian children with rare diseases and barriers to accessing healthcare.
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Single-cell transcriptomic and spatial landscapes of the developing human pancreasCurrent differentiation protocols have not been successful in reproducibly generating fully functional human beta cells in vitro, partly due to incomplete understanding of human pancreas development. Here, we present detailed transcriptomic analysis of the various cell types of the developing human pancreas, including their spatial gene patterns. We integrated single-cell RNA sequencing with spatial transcriptomics at multiple developmental time points and revealed distinct temporal-spatial gene cascades.
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The effects of birth spacing on early childhood development in high-income nations: A systematic reviewThis study aimed to systematically review the literature on the associations between birth spacing and developmental outcomes in early childhood (3–10 years of age). Studies examining the associations between interpregnancy intervals and child development outcomes during and beyond the perinatal period have not been systematically reviewed.
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Rare disease education in Europe and beyond: time to actPeople living with rare diseases (PLWRD) still face huge unmet needs, in part due to the fact that care systems are not sufficiently aligned with their needs and healthcare workforce (HWF) along their care pathways lacks competencies to efficiently tackle rare disease-specific challenges. Level of rare disease knowledge and awareness among the current and future HWF is insufficient.
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Use of administrative record linkage to examine patterns of universal early childhood health and education service use from birth to Kindergarten (age 4 years) and developmental vulnerability in the Preparatory Year (age 5 years) in Tasmania, AustraliaIn Australia, the health and education sectors provide universal early childhood services for the same population of children. Therefore, there is a strong imperative to view service use and outcomes through a cross-sectoral lens to better understand and address the service needs of young children and their families.
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Parental Perspectives on Children’s School Readiness: An Ethnographic StudySchool readiness is a construct used by educators and policy makers to describe a range of abilities that are beneficial for children transitioning to school. The association of socioeconomic disadvantage with developmental vulnerability when children start school is well established. Parents play a crucial role in supporting children’s transition to school and are acknowledged as their child’s first and foremost teacher.
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Aerosolized drug delivery in awake and anesthetized children to treat bronchospasmBronchospasm is a common respiratory adverse event in pediatric anesthesia. First-line treatment commonly includes inhaled salbutamol. This review focuses on the current best practice to deliver aerosolized medications to awake as well as anesthetized pediatric patients and discusses the advantages and disadvantages of various administration techniques.
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Universal Subsidized Continuous Glucose Monitoring Funding for Young People With Type 1 Diabetes: Uptake and Outcomes Over 2 Years, a Population-Based StudyContinuous glucose monitoring (CGM) is increasingly used in type 1 diabetes management; however, funding models vary. This study determined the uptake rate and glycemic outcomes following a change in national health policy to introduce universal subsidized CGM funding for people with type 1 diabetes aged <21 years.
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4CMenB vaccine and its role in preventing transmission and inducing herd immunityVaccination is the most effective method of protecting people from invasive meningococcal disease (IMD). Of all the capsular groups, B is the most common cause of invasive meningococcal disease in many parts of the world. Despite this, adolescent meningococcal B vaccine programs have not been implemented globally, partly due to the lack of evidence for herd immunity afforded by meningococcal B vaccines.