Motor problems, ranging from clumsiness to cerebral palsy, are one of the most common adverse outcomes in children born early. This study will investigate the motor development of children born <30 weeks’ gestation compared with peers born at term from birth to 5 years. We will determine whether early clinical evaluations or neuroimaging in the newborn period can predict later motor impairment at 5 years to be able to identify those who will benefit most from early intervention.
The aim of this proposal is to evaluate a novel therapy option for children with a genetic disorder called mucopolysaccharidosis (MPS). MPS arise from the build up of complex carbohydrates in cells within the body due to the deficiency of an enzyme required for their degradation. By decreasing the synthesis of carbohydrate we can manipulate the level of stored carbohydrate and alleviate the pathology associated with MPS. The novel therapy is based on a chemical modification of glucose that inhib ....The aim of this proposal is to evaluate a novel therapy option for children with a genetic disorder called mucopolysaccharidosis (MPS). MPS arise from the build up of complex carbohydrates in cells within the body due to the deficiency of an enzyme required for their degradation. By decreasing the synthesis of carbohydrate we can manipulate the level of stored carbohydrate and alleviate the pathology associated with MPS. The novel therapy is based on a chemical modification of glucose that inhibits carbohydrate synthesis and is termed substrate deprivation therapy.Read moreRead less
Impact Of A Sleep Intervention In ADHD: Translational Randomised Trial
Funder
National Health and Medical Research Council
Funding Amount
$1,020,595.00
Summary
Up to 50% of children with ADHD experience sleep problems which worsen their ADHD symptoms, behaviour, quality of life and day to day functioning. In a previous trial, we showed that treating sleep problems in children with ADHD improves these outcomes. We now want to know if these benefits can be replicated when general paediatrcians and psychologists deliver the same sleep intervention in community settings.
Frontal-striatal-parietal Activation In Children With ADHD, Combined Type: A Functional Magnetic Resonance Imaging Study
Funder
National Health and Medical Research Council
Funding Amount
$91,750.00
Summary
Attention Deficit Hyperactivity Disorder, combined type (ADHD-CT) is a common neuropsychiatric disorder that has serious consequences for affected children's educational and social development and success in later life. Despite a large investment in research investigating aetiology and therapeutic strategies that arise from these aetiological investigations, ADHD-CT remains poorly understood and it is often viewed with therapeutic pessimism. Understanding the neurobiological basis of ADHD-CT is ....Attention Deficit Hyperactivity Disorder, combined type (ADHD-CT) is a common neuropsychiatric disorder that has serious consequences for affected children's educational and social development and success in later life. Despite a large investment in research investigating aetiology and therapeutic strategies that arise from these aetiological investigations, ADHD-CT remains poorly understood and it is often viewed with therapeutic pessimism. Understanding the neurobiological basis of ADHD-CT is of tremendous importance for the development of more specific and targeted medication and-or psychological treatments and, ultimately, to obtain the best clinical outcome for individual children with ADHD-CT. We have previously examined the function of frontal-striatal-parietal brain networks in adolescent boys with ADHD-CT, showing dysfunction of brain systems important for the control of visuospatial attention. In this project, we aim to examine whether these changes in frontal-striatal-parietal brain function also occur in pre-pubertal 8-12 year-old boys with ADHD-CT. This is important for two major reasons: Firstly, adolescents and young adults examined in previous brain imaging studies of ADHD-CT, including our own, are not truly representative of the core of the disorder, as ADHD-CT has its peak prevalence from 8 to 12 years of age. Secondly, by now comparing pre-pubertal ADHD-CT and healthy control children we can determine whether the changes in brain function we have previously identified represent developmental stage independent brain dysfunction that is characteristic of ADHD-CT.Read moreRead less
Skeletal disease is a major problem for children with mucopolysaccharidoses (MPS). Patients suffer from early onset osteoporosis and osteoarthritis, severely affecting their quality of life. We will evaluate a lentiviral gene therapy vector developed in-house for its capacity to transduce bone, cartilage, synovial and ligament cells in a mouse model of MPS VI. Our goal is to generate high level, sustained expression of the deficient MPS enzyme and alter the course of skeletal disease in MPS.
Improving Child Health Outcomes In Common, High Burden Conditions.
Funder
National Health and Medical Research Council
Funding Amount
$279,895.00
Summary
As medical advances over the past 50 years have helped to reduce traditional childhood illnesses such as infections, behavioural, developmental and mental health problems have increased. These problems affect at least 1 in 5 Australian children yet the vast majority of problems go undetected and untreated. I propose to develop, trial and disseminate evidence-based approaches to common child health problems including mental health and sleep problems.
Tailoring A Brief Sleep Intervention For Autism: A Randomised Controlled Trial
Funder
National Health and Medical Research Council
Funding Amount
$401,475.00
Summary
Up to 86% of children with Autism Spectrum Disorder (ASD) experience behavioural sleep problems which have been shown to be associated with increased core ASD symptoms, increased rates of internalizing and externalizing disorders, and increased parental stress. The “Sleeping Sound” study is a novel behavioural sleep intervention that has shown much promise as a treatment to reduce sleep problems and improve mental health outcomes in children with ASD.
Centre Of Research Excellence (CRE) In Newborn Medicine
Funder
National Health and Medical Research Council
Funding Amount
$2,622,320.00
Summary
Problems around birth are common and can have long-term implications, including into adulthood. Our goal is to improve health outcomes for all newborn babies and their families by determining factors that enhance outcome and assessing the benefits and consequences of new treatments for mothers and babies. We are world leaders in this field and are dedicated to training the next generation of health professionals in the care of newborn babies, in Australia and the rest of the world.
The Effect Of Very Premature Birth On Brain Development
Funder
National Health and Medical Research Council
Funding Amount
$517,975.00
Summary
The neurological outcome of the premature infant is of major importance. Approximately 2,600 premature infants weighing less than 1500 grams are born annually in Australasia. Of the approximate 2,400 survivors between 5-15% will have a more major cerebral palsy, i.e. around 200 children per annum. A greater proportion of 25-50%, i.e., upto 1200 children will have a developmental disability that will adversely affect their school perfomance requiring special assistance or repeating grades. With a ....The neurological outcome of the premature infant is of major importance. Approximately 2,600 premature infants weighing less than 1500 grams are born annually in Australasia. Of the approximate 2,400 survivors between 5-15% will have a more major cerebral palsy, i.e. around 200 children per annum. A greater proportion of 25-50%, i.e., upto 1200 children will have a developmental disability that will adversely affect their school perfomance requiring special assistance or repeating grades. With an increasing number of very prematurely born infants surviving, the absolute number of affected children will continue to rise. Prevention of these disabilities will require an understanding of the cause. The educational and social implications of these high rates of neuro-developmental disability are enormous and the focus of wide international concern. Magnetic Resonance Imaging : It is a major challenge for neonatologists to be able to understand the impact of their therapies and managements on the developing brain. A window into the newborn brain can be seen utilising advanced magnetic resonance imaging techniques in-vivo to investigate these key issues: 1. What is the nature of brain injury in the prematurely born infant? 2. What are the risk factors for brain injury in the prematurely born infant - and are they able to be altered to reduce this risk - e.g. blood pressure management, steroid therapy 3. Is the brain of a prematurely born infant different from that of a full term born infant at TERM equivalent - if so, how is it different? 4. Are there certain postnatal therapies that relate to any alteration in brain structure and chemistry - e.g. postnatal nutrition, modes of ventilation, pharmacological therapies? 5. How does the brain structure relate to function on long term neuro-developmental follow up of our infants at 2 years?Read moreRead less
Optimising Evidence-based, Equitable And Efficient Care For Common Child Health Conditions.
Funder
National Health and Medical Research Council
Funding Amount
$481,156.00
Summary
Care for common conditions such as sleep problems, mental health disorders, and food allergy is often plagued by mismanagement, long wait times, inequitable access, and waste. These factors can worsen child health and quality of life, increase financial and social pressures on families, and deplete the limited health budget. I will develop, implement and evaluate innovative and sustainable solutions to improve care, access to care, and reduce waste in these conditions.