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Research
Transfers of disadvantage across three generations using latent class associations within familiesThere is a large volume of research on the persistence of advantage and disadvantage across generations. Intergenerational studies typically address family resources as independent factors, which ignores how risks cluster together and accumulate over time.
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Preclinical Evaluation of Carfilzomib for Infant KMT2A-Rearranged Acute Lymphoblastic LeukemiaInfants with KMT2A-rearranged B-cell precursor acute lymphoblastic leukemia (ALL) have poor outcomes. There is an urgent need to identify novel agents to improve survival. Proteasome inhibition has emerged as a promising therapeutic strategy for several hematological malignancies. The aim of this study was to determine the preclinical efficacy of the selective proteasome inhibitor carfilzomib, for infants with KMT2A-rearranged ALL.
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Preclinical Evaluation of Carfilzomib for Infant KMT2A-Rearranged Acute Lymphoblastic LeukemiaInfants with KMT2A-rearranged B-cell precursor acute lymphoblastic leukemia (ALL) have poor outcomes. There is an urgent need to identify novel agents to improve survival. Proteasome inhibition has emerged as a promising therapeutic strategy for several hematological malignancies. The aim of this study was to determine the preclinical efficacy of the selective proteasome inhibitor carfilzomib, for infants with KMT2A-rearranged ALL.
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A flexible computational pipeline for research analyses of unsolved clinical exome casesExome sequencing has enabled molecular diagnoses for rare disease patients but often with initial diagnostic rates of ~25-30%. Here we develop a robust computational pipeline to rank variants for reassessment of unsolved rare disease patients. A comprehensive web-based patient report is generated in which all deleterious variants can be filtered by gene, variant characteristics, OMIM disease and Phenolyzer scores, and all are annotated with an ACMG classification and links to ClinVar.
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Does gastrostomy improve the lives of children with severe disability and their families?Approximately 13,000 children in Australia live with moderate to severe intellectual disability.
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Measuring how a disease outbreak could spread in WANick Golding BSc DPhil Honorary Research Fellow Nick.Golding@thekids.org.au Honorary Research Fellow Professor Nick Golding is the UWA Chair in
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Efficacy, Immunogenicity, and Safety Study of Clostridium difficile Toxoid Vaccine in Subjects at Risk for C. difficile InfectionJennifer Peter Kent Richmond RN MBBS MRCP(UK) FRACP Clinical Research Manager Head, Vaccine Trials Group Jennifer.Kent@thekids.org.au Clinical
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A Phase II Randomized, Controlled, Observer-Blinded Study, Conducted to Describe the Immunogenicity, Safety, and Tolerability of a Neisseria Meningitidis Serogroup B Bivalent Recombinant Lipoprotein 2086 Vaccine When Administered to Healthy ToddlersJennifer Peter Kent Richmond RN MBBS MRCP(UK) FRACP Clinical Research Manager Head, Vaccine Trials Group Jennifer.Kent@thekids.org.au Clinical
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Spatial epidemiology of tuberculosisKefyalew Alene BSc, MPH, PhD Head, Geospatial and Tuberculosis 0404705064 Kefyalew.alene@thekids.org.au Honorary Research Fellow Dr Kefyalew Alene

Research
The END RHD CRE: Developing an end game for rheumatic heart disease in AustraliaThe END RHD CRE will undertake a number of projects across several disciplines of research including epidemiology, biomedical sciences; implementation and translation; and understanding the RHD community with a special focus on documenting the experiences of those living with the disease.