A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
a study on AADC Deficiency
AADC Deficiency, AADC, gene therapy, Inborn Errors Amino Acid Metabolism, Dopa Decarboxylase, AAV2-hAADC
Lead Scientists at UCSF
- Nalin Gupta, MD
Nalin Gupta is Chief of the Division of Pediatric Neurosurgery at UCSF, and a Professor in the Departments of Neurological Surgery and Pediatrics. His clinical and research interests are directed towards neuro-oncology and congenital anomalies. He was the lead neurosurgeon at UCSF for a randomized clinical trials examining the effectiveness of fetal surgery for myelomeningocele.
- Krystof Bankiewicz, MD
Dr. Krystof Bankiewicz holds an M.D. from Jagiellonian University in Crakow and a Ph.D., D.Sc. from the Institute of Neurology and Psychiatry in Warsaw, Poland and Professor title by the President of Republic of Poland. Dr. Bankiewicz was a recipient of Fogarty Fellowship and trained at the National Institutes of Neurological Disorders and Stroke at NIH, Bethesda, MD.
- accepting new patients
- Start Date
- Completion Date
- Krzysztof Bankiewicz
- Phase 1 AADC Deficiency Research Study
- Study Type
- Expecting 31 study participants
- Last Updated