Development of therapeutic copper delivery agents for Menkes disease [ 2016 - ]

Research Grant

Researchers: Prof Ashley Bush (Principal investigator)

Brief description Menkes disease does not currently have an effective treatment. The disease is caused by genetic defects that reduce copper transport into the brain and cause mental retardation and death. We have developed drugs that deliver copper into the brain and should cure Menkes disease. We aim to demonstrate that our drugs are effective in mice that have the same genetic defect as patients. Successful results will allow us to begin treating Menkes disease patients to determine if we can cure the disease.

Funding Amount $651,467.00

Funding Scheme Development Grants

Notes Development Grant