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Therapy For CNS Degeneration In MPS Disorders That Targets Both Glycosaminoglycan And Ganglioside Storage.
Funder
National Health and Medical Research Council
Funding Amount
$368,043.00
Summary
Children with seven of the eleven types of mucopolysaccharidosis (MPS) disorders exhibit a profound, irreversible neurological deterioration that manifests in infancy. This results from the continual buildup of undegraded sugar and fat in brain cells. The goal of this proposal is to prevent the accumulation of lipid alone or both lipid and sugar in the brain in order to alter the progression of neurological disease. Treatment will be assessed in mouse models of MPS.
Prophylactic Antibiotics To Prevent Recurrent Lower Respiratory Tract Infections In Children With Neurological Impairment (PARROT) Study
Funder
National Health and Medical Research Council
Funding Amount
$1,210,224.00
Summary
We plan a randomised controlled trial to determine if 12 months of a type of antibiotics (compared to placebo) reduces hospitalisations in children with neurological impairment. Currently this group of children are recurrently hospitalised and some doctors use long term antibiotics but there is no high level evidence for this practice. The study will be undertaken in the UK and Australia and involve 474 children. The study will lead to better clinical care and inform guidelines.
Multi-centre, Multi-disciplinary Study Using A Systems Biology Approach To Investigate Immunomodulation In Children With Acute Wheeze
Funder
National Health and Medical Research Council
Funding Amount
$1,895,107.00
Summary
The concept that immunomodulation using naturally-occurring bacterial agents can treat asthma has reached international prominence. This is backed by strong epidemiologic and clinical trial data. However, detailed knowledge of the immunological mechanisms involved is essential to allow more focused therapeutic agents to be developed. The proposed multi-disciplinary immunomodulation study in 200 children aims to provide this essential information using an advanced systems biology approach.
Randomised Controlled Trial Of The Use Of Nitazoxanide Versus Placebo To Treat Gastroenteritis Among Aboriginal Children
Funder
National Health and Medical Research Council
Funding Amount
$1,324,808.00
Summary
Better treatment strategies are needed for Indigenous children with gastroenteritis. At present, most treatment is supportive only, with little effect on the duration of symptoms. Nitazoxanide appears to have effects on a broad range of pathogens which cause gastroenteritis, but it is not clear whether it will be of benefit in this population. We will compare the speed of symptom resolution in children who receive nitazoxanide with a group who receive a placebo instead.
Nasal CPAP For Very Preterm Infants At Birth: Does It Improve Outcome? A Randomised Controlled Trial
Funder
National Health and Medical Research Council
Funding Amount
$460,604.00
Summary
Neonatal respiratory distress syndrome (RDS) is the major cause of morbidity and mortality in preterm infants. Many of these infants need ventilatory support to keep them alive. In 1996 and 1997, 10,471 infants in Australia and New Zealand needed ventilatory support for a total of 72,544 days. This treatment is a great physical burden for the infants and an enormous emotional stress for their parents. Each day of treatment costs about A$2000 so their hospital treatment costs about $72 million a ....Neonatal respiratory distress syndrome (RDS) is the major cause of morbidity and mortality in preterm infants. Many of these infants need ventilatory support to keep them alive. In 1996 and 1997, 10,471 infants in Australia and New Zealand needed ventilatory support for a total of 72,544 days. This treatment is a great physical burden for the infants and an enormous emotional stress for their parents. Each day of treatment costs about A$2000 so their hospital treatment costs about $72 million a year. Of infants born less than 29 weeks' gestational age, about 40% of the survivors subsequently developed chronic lung disease (CLD). This condition is defined as prolonged dependence on supplementary oxygen therapy. CLD is associated with further costs and increased lung problems and readmissions to hospital in the first year of life. Thus, CLD is an expensive and time-consuming condition that has a high social cost. This project will determine whether treating these very premature babies from birth simply by applying oxygen under a low continuous positive pressure (CPAP) into their nose rather than the present treatment of placing a tube in the windpipe (known as intubation) and ventilation will reduce the incidence and severity of neonatal respiratory distress syndrome and subsequent chronic lung disease. The project will involve 600 babies from different, high quality neonatal intensive care units. Babies who are born at less than 29 weeks' gestation and who show signs of breathing at birth will be randomly allocated to be treated with either nasal CPAP or intubation and ventilation. This project will determine whether CPAP treatment at birth improves survival and reduces the severity of the RDS and subsequent CLD, or has no long term beneficial effect. If the trial is successful, this will be one of the most useful new treatments in neonatal medicine because it is simple to use, easier for the babies, and cheaper than ventilation.Read moreRead less
A Randomised Controlled Trial Of Whole Body Cooling On The Outcome Of Term Infants With Hypoxic Ischaemic Encephalopathy
Funder
National Health and Medical Research Council
Funding Amount
$386,732.00
Summary
The aim of this project is to investigate whether the brain damage caused by a serious lack of oxygen around the time of birth can be prevented or reduced by cooling the baby's temperature to 34C for 72 hours. The consequences, of a lack of oxygen, to the brain, around the time of birth can be devastating. Over 30% of those babies with abnormal brain function soon after birth either die or survive with severe permanent brain damage. There is no specific treatment for these infants. Evidence from ....The aim of this project is to investigate whether the brain damage caused by a serious lack of oxygen around the time of birth can be prevented or reduced by cooling the baby's temperature to 34C for 72 hours. The consequences, of a lack of oxygen, to the brain, around the time of birth can be devastating. Over 30% of those babies with abnormal brain function soon after birth either die or survive with severe permanent brain damage. There is no specific treatment for these infants. Evidence from studies in animals, as well as human adults and a small number of newborn infants, suggests that moderate body cooling started soon after birth in babies with serious abnormal brain function might prevent or reduce brain damage. This project is a multicentre trial, where infants who have suffered from a severe lack of oxygen around birth, are randomised to body cooling to 34C for 72 hours. This will be started as soon as possible after birth at their hospital of birth. If the baby needs to be transported this will be started when the newborn transport team collects the baby for transfer to a newborn intensive care unit. This new treatment will be compared with maintaining the baby's temperature at 37C. This project will investigate a new, simple and pragmatic treatment that might reduce brain damage. If it finds that cooling infants who have been severely deprived of oxygen is an effective and safe treatment, the information will be applicable to any of the very large number of babies around the world who suffer from a serious lack of oxygen around the time of birth.Read moreRead less
The Distinctive Roles Of Tissue Transglutaminase Isoforms In Neuroblastoma
Funder
National Health and Medical Research Council
Funding Amount
$311,567.00
Summary
Neuroblastoma is the commonest solid tumour in early childhood. Neuroblastoma caused by N-Myc oncogene accounts for about one third of the disease and represents a more aggressive subtype with a worse clinical outcome. This project aims to identify factors responsible for N-Myc-induced neuroblastoma initiation and factors sensitizing neuroblastoma cells to anti-cancer drugs, and to provide the basis for clinical trials of a combination therapy in children with neuroblastoma.