AADC Deficiency clinical trials at UCSF
1 research study open to eligible people
AADC deficiency is a rare genetic disorder affecting the brain. UCSF is conducting trials to test new treatments for children with this condition. These trials examine the safety and effectiveness of a gene therapy that delivers the AADC enzyme to the brain.
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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.
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