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
Childhood Diabetes: Prediction, Prevention And Preservation Of Beta Cells
Funder
National Health and Medical Research Council
Funding Amount
$577,189.00
Summary
Childhood onset type 1 diabetes is a severe life-long disease that has a major impact on the child and their family. While studies have attempted to modify the immune system before or after diagnosis, few clinical trials have recruited young children. The overarching goal of this fellowship is to improve the lives of young people with diabetes, through a multifaceted program of ground-breaking research aimed at prediction, prevention and preservation of insulin producing ?-cells in the pancreas.