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A cure for Wolfram syndrome could lead to a cure for diabetes and blindness.

Thursday, June 28, 2012

Drug repurposing

We are looking for drugs that can prevent the progression of neuronal cell death in Wolfram syndrome utilizing different strategies. The challenge we may face is that it may take a long time to get the FDA approval even if we find a drug treatment.

To overcome this challenge, we are interested in a "drug repurposing" strategy. Drug repurposing is the application of known drugs to new diseases. Current NIH director, Dr. Francis Collins, supports this concept and collaborates with the FDA director and major pharmaceutical companies to drive this idea.