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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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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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Late talkers and later language outcomes: Predicting the different language trajectoriesThe aim of the current study was to investigate the risk factors present at 2 years for children who showed language difficulties that persisted
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Infective respiratory syncytial virus is present in human cord blood samples and most prevalent during winter monthsHuman 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 cohortThis cohort study provides new evidence of a disparity between the proportion of the population with rare diseases and their combined health-system costs
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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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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.
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Increased heterogeneity of airway calibre in adult rats after hypoxia-induced intrauterine growth restrictionThe rat model demonstrates that intrauterine growth restriction leads to a more heterogeneous distribution of airway lumen calibre in adulthood
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Investigating self-efficacy, disease knowledge and adherence to treatment in adolescents with cystic fibrosisadolescents with cystic fibrosis are often sub-optimal with adherence to prescribed treatement. We aim to investigate multiple factors with patient and parents
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Early introduction of food reduces food allergy – Pro and ConWhen an infant is developmentally ready, a variety of nutritious foods should be introduced including the ‘more allergenic’ foods during infancy