PND Research
The 2012-2014
Research Grant Program
Awards up to $107,500 to Research On Behalf of Pediatric Neurotransmitter Diseases
I. Recipient: Umrao Monani , Ph.D., Associate Professor of Pathology and Cell Biology in Neurology Columbia University Medical Center, New York
Award: $12,500 Phase 1 "Generation and characterization of a mouse model of AADC Deficiency".
II. Recipient: Krystof Bankiewicz, M.D., Ph.D., Director Bankiewicz Laboratory, Professor of Neurosurgery and Neurology Parkinson's Disease Clinic and Research Center, Kinetics Foundation Chair in Translational Research, University of California, San Francisco
Award: $40,000 Regulatory support in preparation of a clinical study of AAV2—hAADC Gene Therapy in AADC deficient children.
III. Toni Pearson MD, MBBS, Assistant Professor of Neurology, Divisions of Movement Disorders and Child Neurology, Icahn School of Medicine at Mount Sinai, New York
Award: $30,000 "To develop the clinical trial protocol for AADC gene therapy and to characterize the natural history of AADC deficiency".
IV. Recipient: Ellen J. Hess Ph.D., Departments of Pharmacology and Neurology, Emory University School of Medicine, Atlanta
Award: $25,000 "Analysis of the TH mouse model to determine the dopamine receptor subtype(s) and signaling defects that contribute to abnormal movements"
PND Research Grant Program
For information about the PND Research Grant Program.
Past Research Grant Recipients
Past Research Grant Recipients
Research Grant Review Committee
Committee members and contact information for the research grant review program
Stem Cells request
Seeking patients with GTPCH, TH, and AADC for dystonia stem cell research project.For detailed information about clinical trials and how drugs/treatments are developed please visit: