Neonatal Cranial Ultrasound: Safety, Knowledge And Practice Of Prolonged Or Repeated Exposure To Acoustic Waves
Funder
National Health and Medical Research Council
Funding Amount
$255,055.00
Summary
Ultrasound is routinely used for diagnosis of brain lesions amongst newborns. Animal studies have shown that ultrasound can sometimes be harmful to the brain. We currently have very little information on the characteristics of neonatal cranial scans. This project will investigate current knowledge of safety and training via a nationwide survey, analyse the elements of 'typical' scans and measure brain surface heating after ultrasound amongst neonates.
Mucopolysaccharidoses (MPS) are a related group of 11 debilitating genetic disorders affecting children. They result from a reduction or total deficiency of an enzyme required for the removal of carbohydrate structures called glycosaminoglycans (gags). Gag degradation occurs inside the cell in specific organelles termed lysosomes and in the absence of the appropriate enzyme, undegraded gag accumulates in the cell. This leads to a range of clinical symptoms and multiple tissue failure. Symptoms c ....Mucopolysaccharidoses (MPS) are a related group of 11 debilitating genetic disorders affecting children. They result from a reduction or total deficiency of an enzyme required for the removal of carbohydrate structures called glycosaminoglycans (gags). Gag degradation occurs inside the cell in specific organelles termed lysosomes and in the absence of the appropriate enzyme, undegraded gag accumulates in the cell. This leads to a range of clinical symptoms and multiple tissue failure. Symptoms common to more than one MPS type include mental deterioration, blindness, abdominal organ enlargement and bone growth problems leading to short stature and bone loss. My laboratory has had a long-term interest in developing treatment for MPS and our research led to the clinical implementation of enzyme replacement therapy (ERT) for MPS VI in 2005. While providing the first effective, multi-tissue treatment for MPS, our research showed that several tissues were not responsive to ERT. These are the brain, cartilage and cornea, thus children on ERT regimens will still suffer from mental retardation, arthritis and blindness. With the goal of treating these particular tissues we have developed a new approach to MPS therapy called substrate deprivation therapy (SDT). Instead of adding back the missing enzyme, SDT acts by decreasing gag production which in turn reduces the level of accumulated gag in cells. SDT results in the correction of MPS cells in culture and reduces several key clinical symptoms in the mouse model of MPS IIIA. In this proposal we will extend our research to evaluate the effect of SDT on brain and bone-joint pathology. Evaluation of efficacy will take place in the MPS VII mouse which exhibits both brain and bone disease and in a new model of MPS IVA developed specifically for this study which exhibits a joint pathology unique amongst the MPS disorders.Read moreRead less
I am a psychologist whose research program aims to improve the long-term well-being for children born very small or immature. My research focuses on 1) determining the nature and severity of cognitive and behavioural problems faced by children born very small-immature, 2) investigating how these problems are associated with brain injury and alterations to brain development, and 3) assessing the effectiveness of clinical interventions which aim to reduce complications and enhance development.
Improving Treatment And Outcomes Of Congenital Hearing Loss At The Population Level
Funder
National Health and Medical Research Council
Funding Amount
$265,138.00
Summary
Around 600 Australian babies are born with congenital hearing loss each year. Despite great advances in early diagnosis and treatment, affected children have language and other skills well below what they can potentially achieve. My research will a) examine trends in congenital hearing loss outcomes, b) enrich existing data with novel genetic and clinical data, to enable early prediction of future outcomes to direct management, and c) discover ways to prevent hearing loss progression.
Understanding Childhood Speech And Language Disorders: What Goes Wrong, When And Why It Matters?
Funder
National Health and Medical Research Council
Funding Amount
$466,849.00
Summary
My vision for the next five years is to improve greatly the diagnosis, prevention and management of childhood communication impairments. Making a difference for these children and their families means improving our understanding of how speech and language develops, what goes wrong, when, and why it matters. My research program will target each of these topics and outline a plan for translation, that is how to get the new knowledge and best evidence into practice.
I am a perinatal paediatrician undertaking clinically-focussed research on brain development, brain disorders, brain therapies, neurodevelopmental outcomes and the development, application and evaluation of new technology to clinical problems.
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
DETERMINING THE MECHANISMS LEADING TO LONG-TERM IMPAIRMENT IN VERY PRETERM CHILDREN: THE VIBeS LONGITUDINAL STUDY.
Funder
National Health and Medical Research Council
Funding Amount
$1,061,733.00
Summary
Approximately 50% of children born very preterm will develop significant problems. The objective of this project is to determine the mechanisms leading to these problems. We will do this by assessing at school-age a unique and valuable cohort of very preterm infants (VIBeS cohort) who had state-of-the-art brain scans shortly after birth and neurobehaviour assessments across early childhood. This school-age follow-up will involve repeat brain scans and detailed neuropsychological assessments.