Surgical Management Of The Pulmonary Circulation In Children
Funder
National Health and Medical Research Council
Funding Amount
$114,328.00
Summary
Congenital disorders of the lung circulation are rare. These children often present during infancy with symptoms of heart failure and require surgery to correct these defects. Without surgery, the prognosis of these conditions are poor. Our understanding of these conditions are limited. The proposed study aims to review all patients who underwent surgical repair of abnormalities of lung arteries and veins at the Royal Children’s Hospital.
The Role Of Emerging Gastrointestinal Viruses In The First Two Years Of Life: A Birth Cohort Study
Funder
National Health and Medical Research Council
Funding Amount
$392,534.00
Summary
Gastro illnesses are common and potentially serious early in life. We are aiming to find out more about these illnesses by following ~140 children from birth to their second birthday, with parents collecting a dirty nappy swab every week. Our laboratory will test these samples for a wide range of known and new gastro viruses, and we will also be looking for, as yet, undiscovered viruses. This information will allow us to document the burden of these illnesses in young children and their families ....Gastro illnesses are common and potentially serious early in life. We are aiming to find out more about these illnesses by following ~140 children from birth to their second birthday, with parents collecting a dirty nappy swab every week. Our laboratory will test these samples for a wide range of known and new gastro viruses, and we will also be looking for, as yet, undiscovered viruses. This information will allow us to document the burden of these illnesses in young children and their families.Read moreRead less
In Vitro And In Vivo Assessment Of The Funhaler -an Innovative Therapeutic Device For Children
Funder
National Health and Medical Research Council
Funding Amount
$472,750.00
Summary
Aerosol therapy is the most effective form of treatment for children with respiratory diseases such as asthma. While optimising aerosol delivery systems has an important role in increasing the efficacy of asthma therapy, ensuring patient compliance is often the most difficult part of the clinician's role, particularly in the paediatric age group. An innovative small volume spacer device (Funhaler) developed by a West Australian company (InfaMed, Ltd) may help overcome this problem. The Funhaler ....Aerosol therapy is the most effective form of treatment for children with respiratory diseases such as asthma. While optimising aerosol delivery systems has an important role in increasing the efficacy of asthma therapy, ensuring patient compliance is often the most difficult part of the clinician's role, particularly in the paediatric age group. An innovative small volume spacer device (Funhaler) developed by a West Australian company (InfaMed, Ltd) may help overcome this problem. The Funhaler incorporates a spinning toy attached to the outside of the spacer. The toy is activated when the patient breathes through the spacer. The device has been designed to encourage children to co-operate when their asthma therapy is being delivered. The Funhaler is currently in the late development stage. We propose, firstly, to carry out in vitro assessments of drug delivery from the Funhaler compared to the two most widely available small volume spacers: the Aerochamber Plus (Trudell, Canada) and the Breath-A-Tech (Scott-Dibben, Australia). These assessments will be carried out to meet the standards of regulatory bodies worldwide (including the FDA). Secondly, we propose to perform extensive in vivo studie: filter studies to assess drug delivery to the patient; deposition studies to measure drug deposition in the lungs; and a pilot clinical trial to assess the efficacy of the device during medium to long-term use in children aged 2-8 years.Read moreRead less
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
A Study To Determine The Effects Of Heparin/ Low Molecular Weight Heparin In Neonates And Children.
Funder
National Health and Medical Research Council
Funding Amount
$193,000.00
Summary
Blood clots in newborns and children are becoming a more common problem. This is because many children with major illnesses are now surviving due to the remarkable advances in medical and surgical care. Blood clots in children can have devastating long term effects. Little is known about the best way to treat blood clots in children and most treatments are just extrapolated from adult treatment guidelines. This is unlikely to be the best treatment as the type and place of blood clots in children ....Blood clots in newborns and children are becoming a more common problem. This is because many children with major illnesses are now surviving due to the remarkable advances in medical and surgical care. Blood clots in children can have devastating long term effects. Little is known about the best way to treat blood clots in children and most treatments are just extrapolated from adult treatment guidelines. This is unlikely to be the best treatment as the type and place of blood clots in children are very different to adults. In addition, the blood clotting system in children is very different to that in adults. This is especially true for newborns. Over the last four years we have established the largest clinical treatment program for children with blood clots in Australia, and have completed the preliminary work that will enable us to now study a number of aspects of the treatment for blood clots in children. This project will specifically examine heparin and low molecular weight heparin which are the most commonly used antithrombotic (anti blood clot) drugs in children. We will determine the effect of age on the mechanism of action, the optimal drug level for treatment, the frequency of the most common side effect of heparin and do some preliminary work to determine alternative treatment options. Our study will provide the basis for more appropriate use of these drugs in children, which will improve the success of therapy and reduce the risk of complications, ultimately improving the survival and quality of life for sick children affected by blood clots.Read moreRead less
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.
The Centre For Research In Childhood Early Respiratory Disease
Funder
National Health and Medical Research Council
Funding Amount
$2,621,023.00
Summary
The Centre for Research in Childhood Early Respiratory Disease is dedicated to the prevention of lung disease in young children with chronic respiratory disease. We aim to better understand the process of lung disease to identify predictors of disease and to treat it better. We will investigate the psychosocial effects of early interventions to better manage families. We will facilitate collaborations to provide best practice guidance and will train the next generation of doctors and researchers
Understanding The Factors Governing Susceptibility And Outcome In Childhood Infection
Funder
National Health and Medical Research Council
Funding Amount
$276,122.00
Summary
This research seeks to understand why a minority of children are prone to severe and often life-threatening infections and inflammation. It focusses on infections both in preterm infants and in later childhood, which may also be relevant to understanding atherosclerosis. I am also interested in improving the health of recently arrived refugees, by conducting research that allows the development of evidence-based health interventions and developing national policy on refugee health.
A Randomized Controlled Trial Of Effects Of Early Life Exposure To General Anaesthesia On Neurobehavioural Outcomes In Children With Cystic Fibrosis (CF)
Funder
National Health and Medical Research Council
Funding Amount
$587,240.00
Summary
Anaesthesia permits surgeries and other interventional procedures that benefit the health of children to be performed painlessly and non-traumatically. This study will provide critical information about whether the drugs used commonly for general anaesthesia represents a risk to very young children in terms of their neurobehavioural development.
Limiting Tuberculosis Transmission And Improving The Care Of Affected Children
Funder
National Health and Medical Research Council
Funding Amount
$412,419.00
Summary
Tuberculosis (TB) is the biggest infectious disease killer on the planet. Drug-resistant TB poses a particular challenge in the Asia-Pacific region. My research will assist Australia to progress towards domestic TB elimination. It will improve the management of “difficult to treat” cases and help to safeguard the public against ongoing TB transmission. Work in neighbouring countries will help to contain the spread of drug-resistant TB and protect vulnerable young children.