AADC Deficiency clinical trials at UCSF

1 research study open to eligible people

Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
open to eligible people ages 24 months and up
The overall objective of this study is to determine the safety and efficacy of AAV2-hAADC delivered to the substantia nigra pars compacta (SNc) and ventral tegmental area (VTA) in children with aromatic L-amino acid decarboxylase (AADC) deficiency.
San Francisco, California and other locations

Our lead scientists for AADC Deficiency research studies include Nalin Gupta, MD.

Last updated: July 9, 2026

Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients