The Neurodegenerative Diseases Research Laboratory (NDRL) is developing a cure for a form of Batten disease, an inherited disorder in children that causes progressive loss of all brain functions and vision loss and ultimately results in death. Using a dog model of Batten disease, the NDRL has developed a treatment that completely preserves vision in affected dogs. The lab is now testing whether brain structure and function can be preserved using the same approach to treatment. Our goal is to make the treatments that are working so well in the dogs available to children with Batten disease. Translating the treatment to human application will be very expensive, requiring the design, regulatory approval, and conduct of human clinical trials. We are seeking funds to enable us to develop and conduct these trials so that the treatment we have developed will be available to children suffering from Batten disease.
For more information on Dr. Katz’s NDRL laboratory, visit his research website.