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
Respiratory failure at birth is a major cause of death and disease in newborn infants. At birth the airways must be cleared of liquid to allow the inhalation of air, but, little is known about the process of lung aeration, because it has not been possible to observe or measure it. We have developed imaging and analytical techniques to observed and measure lung aeration. We will determine ventilation procedures that promote uniform lung aeration and minimise lung injury in ventilated infants.
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.
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