2013 Cure Dystonia Now Foundation Grant

We are extremely pleased to have begun to fund our first research project in May 2013:


“Promising Treatments for Dystonia: Preclinical Testing”


Ellen J. Hess, Ph.D.
Professor of Pharmacology and Neurology
Emory University School of Medicine


Therapies for dystonia are largely unsatisfactory or palliative. Therefore, there is a tremendous need for new drugs that are truly effective for the treatment of the dystonias. Although drug discovery for dystonia was unimaginable just a few years ago, this is, fortunately, now possible in light of major advances in dystonia research. In the upcoming months, we will be performing preclinical testing of several FDA-approved drugs that have promise as treatments for DYT1 dystonia. These drugs were first identified using cell-based models to find drugs with the potential to correct cell abnormalities associated with DYT1 dystonia. We will now test these drugs in mouse models of dystonia. Testing in animals will help determine if the drugs have antidystonic properties; testing in animals is also necessary to advance a drug to clinical trial.  The primary, immediate goal of this study is to identify 1-2 promising candidates that could be used for clinical trials in patients with DYT1 or other forms of dystonia. A secondary goal is to identify possible unexpected actions of the drugs that may actually facilitate the antidystonic effects or may contribute to unwanted side-effects.  This information will be useful for designing novel drugs for dystonia, which is one of the long-term goals of this study.


  • Your gift to Cure Dystonia Now will fund research toward more and/or improved treatments, and ultimately a cure, for Dystonia. We welcome and appreciate donations of any amount. Donations to Cure Dystonia Now are tax deductible as allowable by law.
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Cure Dystonia Now
201 Old Country Road, Suite 205
Melville, NY 11747
Office: (516) 584-4156