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: