michaeljfox.org/grant/pheny...
Objective/Rationale:
In our previous research, we have discovered that a drug called phenylbutyrate can turn on a protective gene in the brain and can prevent Parkinson’s disease (PD) in models that are genetically programmed to get the disease. The drug works by transferring abnormal proteins from brain cells into the bloodstream. We now want to see if the drug works in patients with Parkinson’s in the same way. If it does, the drug may be able to stop the progression of Parkinson’s disease.