AADC Deficiency clinical trials at UCSF
1 research study open to eligible people
AADC deficiency is a disorder that disrupts normal brain chemical production. UCSF is researching a gene therapy to see if it is safe and works well in young patients. The study uses different doses to find the most effective treatment.
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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
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