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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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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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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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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
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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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Mental health problems among 4–17-year-olds with hearing problems: results from a nationally representative studyClinicians caring for children and young people with hearing problems should be alert for heightened risk of specific mental health problems based on age and the nature of hearing problems.
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Initiating an undiagnosed diseases program in the Western Australian public health systemWe describe the initiation and summary outcomes of a public health system approach for complex undiagnosed patients- the Undiagnosed Diseases Program - WA
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Evaluation of age-dependent treatment strategies for children and young adults with pineoblastoma: Analysis of pooled European Society for Paediatric Oncology (SIOP-E) and US Head Start dataPineoblastoma is a rare pineal region brain tumor. Treatment strategies have reflected those for other malignant embryonal brain tumors.
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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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In vitro drug susceptibility of two strains of the wildlife trypanosome, Trypanosoma copemani: A comparison with Trypanosoma cruziCompared the in vitro susceptibility of two strains of Trypanosoma copemani and one strain of T. cruzi against drugs that show trypanocidal activity