Substrate deprivation therapy for MPS disorders [ 2004 - 2006 ]

Also known as: A new treatment for an inherited metabolic disorder

Research Grant

[Cite as]

Researchers: Dr Sharon Byers (Principal investigator)

Brief description 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.

Funding Amount $AUD 209,250.00

Funding Scheme NHMRC Project Grants

Notes Standard Project Grant

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