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.
AADC Deficiency
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.
A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
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University of California San Francisco, Benioff Children's Hospital, San Francisco, California, United States, 94143
Nationwide Children's Hospital, Columbus, Ohio, United States, 43205
The Ohio State University Medical Center, Columbus, Ohio, United States, 43221
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24 Months to
ALL
No
Krzysztof Bankiewicz,
Krystof Bankiewicz, MD, PhD, STUDY_DIRECTOR, OSU Professor of Neurological Surgery
2031-07