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Showing results for "clinical trials"
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Improving clinical trial readiness to accelerate development of new therapeutics for Rett syndromeRett syndrome is associated with severe functional impairments and many comorbidities, each in urgent need of treatments. Mutations in the MECP2 gene were identified as causing Rett syndrome in 1999. Over the past 20 years there has been an abundance of preclinical research with some studies leading to human clinical trials.
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A randomised-controlled trial of a behavioural intervention for optimising social and communication development in newborns at increased likelihood of autism spectrum disordersAndrew Kandice Matt Melissa Videos Whitehouse Watch and listen to Andrew Varcin Cooper Licari PhD M.Psych (Clinical), PhD BCA Marketing, BSc
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Beyond Seizures as an Outcome Measure: A Global Severity Scoring System for CDKL5 Deficiency DisorderCDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy (DEE) associated with multiple impairments and comorbidities. Outcome measures for disease-modifying clinical trials for DEEs should measurably capture a spectrum of caregiver priorities and be externally validated.
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Multi-site validation of a suite of clinical outcome measures for clinical trial readiness in the CDKL5 Deficiency DisorderHelen Jenny Peter Leonard Downs Jacoby MBChB MPH BApplSci (physio) MSc PhD BA (Hons) MSc Principal Research Fellow Head, Child Disability
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Active surveillance of 2017 seasonal influenza vaccine safety: An observational cohort study of individuals aged 6 months and older in AustraliaAusVaxSafety surveillance demonstrated comparable and expected safety outcomes for the 2017 quadrivalent inactivated influenza vaccine brands used in Australia
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Novel coenzyme Q6 genetic variant increases susceptibility to pneumococcal diseaseAcute lower respiratory tract infection (ALRI) remains a major worldwide cause of childhood mortality, compelling innovation in prevention and treatment. Children in Papua New Guinea (PNG) experience profound morbidity from ALRI caused by Streptococcus pneumoniae. As a result of evolutionary divergence, the human PNG population exhibits profound genetic variation and diversity. To address unmet health needs of children in PNG, we tested whether genetic variants increased ALRI morbidity.
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Mandating COVID-19 vaccinations for children: Attitudes of Western Australian parentsAustralian governments have used vaccine mandates to drive high uptake of routine childhood vaccines and adult Coronavirus Disease 2019 (COVID-19) and influenza vaccines. We sought to understand the attitudes of Western Australian parents regarding mandating COVID-19 vaccines for children, interviewing 44 parents of children aged up to 18 years between May and December 2021. Transcripts were analysed to ascertain parents' attitudes and sources of reasoning.
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Calling Future Grandparents-Further Efforts Required to Increase Human Papillomavirus Vaccination Use in AdolescenceChristopher Blyth MBBS (Hons) DCH FRACP FRCPA PhD Centre Head, Wesfarmers Centre of Vaccines and Infectious Diseases; Co-Head, Infectious Diseases
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Consensus guidelines for the diagnosis and management of cryptococcosis and rare yeast infections in the haematology/oncology setting, 2021Cryptococcosis caused by the Cryptococcus neoformans-Cryptococcus gattii complex is an important opportunistic infection in people with immunodeficiency, including in the haematology/oncology setting.
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Prospective characterisation of SARS-CoV-2 infections among children presenting to tertiary paediatric hospitals across Australia in 2020: A national cohort studyTo present Australia-wide data on paediatric COVID-19 and multisystem inflammatory syndromes to inform health service provision and vaccination prioritisation. Design Prospective, multicentre cohort study. Setting Eight tertiary paediatric hospitals across six Australian states and territories in an established research surveillance network - Paediatric Active Enhanced Disease (PAEDS).