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Determining risk features for medulloblastoma in the molecular eraNick Gottardo MBChB FRACP PhD Head of Paediatric and Adolescent Oncology and Haematology, Perth Children’s Hospital; Co-head, Brain Tumour Research
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The Feasibility of Personalized Endpoints in Assessing Treatment Outcomes for Rare Diseases: A Pilot Study of Goal Attainment Scaling in SCN2A-AssociatedFor individuals living with rare neurodevelopmental disorders, particularly those who are at the most severe end of the spectrum, standardized outcome measures may lack the sensitivity to capture small but meaningful changes.
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Awake Supraglottic Airway Placement in Pediatric Patients for Airway Obstruction or Difficult Intubation: Insights From an International Airway Registry (PeDI)Small case series have described awake supraglottic airway placement in infants with significant airway obstruction and difficult intubations. We conducted this study to determine outcomes when supraglottic airways were placed in awake children enrolled in the international Pediatric Difficult Intubation Registry including success of ventilation, success of tracheal intubation, and complications.
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A cross-sectional audit of the Australian community food environment highlights the prominent role of chain food outletsFood retail and food service outlets can be part of a chain, or independently operated. Chain food outlets are likely to have the most influence over community food environments but have not been routinely identified in studies which map and monitor access to food, highlighting an important knowledge gap. This study aimed to identify the food retail and food service outlets present within metropolitan Perth, including presence of chain and independent food outlets; and examine differences across local governments.
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The future of paediatric obstructive sleep apnoea assessment: Integrating artificial intelligence, biomarkers, and moreAssessing obstructive sleep apnoea in children involves various methodologies, including sleep studies, nocturnal oximetry, and clinical evaluations. Previous literature has extensively discussed these traditional methods.
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Are assisted reproductive technology pregnancies more likely to be exposed to teratogenic medication? A whole-population studyAssisted reproductive technology (ART) pregnancies are at greater risk of birth defects than non-ART pregnancies. Teratogenic medication exposure is a potential cause of birth defects that has not been compared between ART and non-ART pregnancies.
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Compatibility of pentoxifylline injection with syringe and inline filtersTobias Strunk MD, PhD, FRACP Head, Neonatal Health tobias.strunk@thekids.org.au Head, Neonatal Health Clinical Professor Tobias Strunk is a
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Protocol to implement and evaluate a culturally secure, strength-based, equine-assisted learning program, "Yawardani Jan-ga" (horses helping)Australian Aboriginal people experience stressors from inequalities across crucial social determinants, including deep and entrenched disadvantage and exclusion. The impact of unaddressed historical issues is pervasive and intergenerational. The disproportionate rates of Aboriginal youth suicide, juvenile detention and imprisonment highlight the inadequacy of existing social and emotional wellbeing programs and services for Aboriginal children and young people.
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The effectiveness of maternal pertussis vaccination for protecting Aboriginal and Torres Strait Islander infants against infection, 2012–2017: a retrospective cohort studyTo evaluate the effectiveness of maternal pertussis vaccination for preventing pertussis infections in Aboriginal and Torres Strait Islander infants under seven months of age.
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FDA Patient-Focused Drug Development Guidances: Considerations for Trial Readiness in Rare Developmental and Epileptic EncephalopathiesDevelopmental and epileptic encephalopathies (DEE) are rare, often monogenic neurodevelopmental conditions. Most affected individuals have refractory seizures. All have multiple severe impairments which can be as life-limiting as or more limiting than the seizures themselves. Mechanism- and gene-targeted therapies for these individually rare, genetic conditions hold hope for treatment, amelioration of disease expression, and even cure.