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Research
RD-RAP: Beyond rare disease patient registries, devising a comprehensive data and analytic frameworkWe introduce and describe the concept of a Rare Disease Registry and Analytics Platform
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No detectable effects of acute tryptophan depletion on short-term immune system cytokine levels in healthy adultsThe acute tryptophan depletion condition did not result in significant changes to cytokine concentrations for the entire study sample
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Mental wellbeing in non-ambulant youth with neuromuscular disorders: What makes the difference?Mental wellbeing was independently associated with academic achievement and perceived family support but not with physical health variables
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Transcriptional landscape of Mycobacterium tuberculosis infection in macrophagesA comprehensive in depth gene expression/regulation profile in Mycobacterium tuberculosis-infected macrophages
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Macrolides in children with community-acquired pneumonia: Panacea or placebo?Pneumonia, most often caused by a respiratory virus, is common in childhood.
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Contemporary survival endpoints: An international diffuse intrinsic pontine glioma registry studyThis study defines PFS and OS, and is the first describe post-progression survival in a large cohort of children with DIPG.
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Gestational age and school achievement: A population studyWe examined gestational age from preterm to post-term against a national minimum standard for academic achievement in population data.
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Does alcohol outlet density differ by area-level disadvantage in metropolitan Perth?This ecological study examines the distribution of liquor licences in Perth, Australia, and whether discrepancies in the distribution of retail land-uses could account for a socio-economic gradient.
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A Phase I Study of the CDK4/6 Inhibitor Ribociclib (LEE011) in Pediatric Patients with Malignant Rhabdoid Tumors, Neuroblastoma, and Other Solid TumorsIn this. i study the MTD and RP2D, safety, PK, and preliminary activity of single-agent ribociclib were investigated in patients with neuroblastoma.
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Prevalence of microcephaly in an Australian population-based birth defects register, 1980-2015We identified a high proportion of cases without known cause, highlighting the need for clinicians to carefully investigate all possibilities, including emerging infections.