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Research

A Phase I Study of the CDK4/6 Inhibitor Ribociclib (LEE011) in Pediatric Patients with Malignant Rhabdoid Tumors, Neuroblastoma, and Other Solid Tumors

In this. i study the MTD and RP2D, safety, PK, and preliminary activity of single-agent ribociclib were investigated in patients with neuroblastoma.

Research

Infective respiratory syncytial virus is present in human cord blood samples and most prevalent during winter months

Human respiratory syncytial virus (RSV) remains the most common cause of severe lower respiratory tract disease amongst infants, and continues to cause annual epidemics of respiratory disease every winter worldwide.

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The collective impact of rare diseases in Western Australia: An estimate using a population-based cohort

This cohort study provides new evidence of a disparity between the proportion of the population with rare diseases and their combined health-system costs

Research

Prevalence of microcephaly in an Australian population-based birth defects register, 1980-2015

We identified a high proportion of cases without known cause, highlighting the need for clinicians to carefully investigate all possibilities, including emerging infections.

Research

Investigating self-efficacy, disease knowledge and adherence to treatment in adolescents with cystic fibrosis

adolescents with cystic fibrosis are often sub-optimal with adherence to prescribed treatement. We aim to investigate multiple factors with patient and parents

Research

Toxoplasma Modulates Signature Pathways of Human Epilepsy, Neurodegeneration & Cancer.

disease-deconvolution" identified associations between the parasite-brain interactions and epilepsy, movement disorders, Alzheimer's disease, and cancer.

Research

Probabilistic linkage of national immunisation and state-based health records for a cohort of 1.9 million births to evaluate Australia’s childhood immunisation program

To describe the process for assembling a linked study that will enable the conduct of population-based studies related to immunisation and immunisation policy.

Research

The continuity and duration of depression and its relationship to non-suicidal self-harm and suicidal ideation and behavior in adolescents 12–17

Overlap between non-suicidal self-harm and suicidal ideation and behavior in young people with both symptom continuity and symptom duration implicated in this association

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Contemporary survival endpoints: An international diffuse intrinsic pontine glioma registry study

This study defines PFS and OS, and is the first describe post-progression survival in a large cohort of children with DIPG.

Research

Increased heterogeneity of airway calibre in adult rats after hypoxia-induced intrauterine growth restriction

The rat model demonstrates that intrauterine growth restriction leads to a more heterogeneous distribution of airway lumen calibre in adulthood