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Prenatal ambient heat exposure and neurodevelopment: A scoping review of human and animal researchAmbient heat exposure during pregnancy is associated with adverse outcomes, and adverse neurodevelopmental outcomes are an emerging concern. This scoping review synthesises human and animal evidence on the association between prenatal ambient heat exposure and poor neurodevelopmental outcomes.
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Children's Anxiety in the Perioperative Environment: A Qualitative Exploration With Children, Parents and Staff at a Tertiary Paediatric HospitalPerioperative anxiety is a common and distressing aspect of anaesthesia for many children, resulting in management challenges at the time of anaesthesia and potential physical and psychological adverse outcomes. We conducted this qualitative phenomenological study to explore the perspectives of children, parents and staff on perioperative anxiety in our institution. Planned recruitment was 20 each of children who had undergone elective anaesthesia, their parents and staff.
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Fc-Engineered B7-H3 Antibody with Prolonged Serum Half-Life for Enhanced Cancer TherapyMonoclonal antibodies are revolutionizing the landscape of current cancer treatment, bringing hope to patients with incurable cancers. B7-H3 (CD276) is an attractive therapeutic target for antibody-based therapy due to its low or absent expression in normal tissues and high expression in various types of tumors, including prostate cancer, pancreatic cancer, and high-mortality esophageal squamous cell carcinoma (ESCC). In recent years, various B7-H3-targeting antibodies have been developed for cancer treatment, with a few making their way to clinical trials.
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Characterising commensal and pathogenic staphylococcal interactions with neonatal and adult bloodThe abundant skin commensal, Staphylococcus epidermidis, is the leading cause of late-onset sepsis (LOS) in preterm infants but rarely causes infections in term infants and adults. Staphylococcal virulence mechanisms and the role of the preterm immune responses in driving these life-threatening infections remain poorly understood.
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Association Between First-Trimester Medication Exposure in Pregnancy and Congenital Anomalies: A Scoping Review of Cohorts, Exposure, Trimester and Congenital Anomaly DefinitionsThe risk of congenital anomalies following first-trimester medication exposure is an important indicator of medication safety during pregnancy. Retrospective cohort studies using routinely collected data are commonly used to assess this risk, yet methodological inconsistencies-such as how cohorts, exposures, timings and outcomes are defined-can compromise reproducibility and validity. This scoping review examined the methodologies used in retrospective cohort studies assessing the association between first-trimester prenatal medication exposure and congenital anomalies.
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Six cases of ENPP1 pathogenic variants causing autosomal recessive hypophosphatemic rickets type 2 and generalized arterial calcification of infancyAutosomal recessive hypophosphatemic rickets type 2 (ARHR2) and generalized arterial calcification of infancy (GACI) occur secondary to biallelic ectonucleotide pyrophosphate/phosphodiesterase 1 (ENPP1) loss-of-function pathogenic variants. GACI is a life-threatening condition, often presenting in the neonatal period with heart failure and hypertension, caused by calcification of the media in large- and medium-sized arteries.
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What is it like living with X-linked hypophosphatemia?: results from an Australian consumer surveyX-linked hypophosphatemia (XLH) is a rare, X-linked dominant condition with a high burden of both physical and psychosocial disease. This study aimed to describe the experience and burden of disease for children and adults living with XLH in Australia by inviting affected individuals and their carers to complete an online questionnaire. Of the 46 responses, half were completed by a person with XLH, and half by carers. Thirty percent were male, 33% were aged less than 18 yr.
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Factors Associated With Rare Pediatric Cancer Trial Enrollment: A Report From the Children's Oncology Group Rare Tumors CommitteeOver 90% of US children with cancer are treated at Children's Oncology Group (COG) centers, which seek to maximize enrollment in therapeutic and biobanking studies. Rare cancers have demonstrated lower than expected COG enrollment. We evaluated trends in COG rare cancer enrollment compared to US incidence from Surveillance, Epidemiology, and End Results (SEER) registries, examining the impact of COG therapeutic trials and Project:EveryChild, a cancer biobank/registry.
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Parents’ Work–Family Conflict and Children’s Emotional Well-Being: The Mediating Role of Parenting BehaviorsDespite growing evidence that parental work–family conflict (WFC) affects children’s emotional well-being, little is known about the multiple pathways underlying such effects. This study examines the association between parental WFC and children’s emotional well-being and the potential mediating role of parenting behaviors in this process.
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The United Nations convention on rare diseases—A framework for research prioritizationCitation: Junaid M, Downs J, Groza T, Lassmann T, Baker S, et al. The United Nations convention on rare diseases—A framework for research