A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
NCT02852213
·
clinicaltrials.gov ↗
PHASE1
Phase
RECRUITING
Status
42
Enrollment
OTHER
Sponsor class
Conditions
AADC Deficiency
Interventions
DRUG:
AAV2-hAADC
Sponsor
Krzysztof Bankiewicz
Collaborators
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