Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes from the PD-1101 Trial

Safety of AADC Gene Therapy for Moderately Advanced Parkinson Disease: Three-Year Outcomes from the PD-1101 Trial

Background and Objectives To report final, 36-month safety and clinical outcomes from the PD-1101 trial of NBIb-1817 (VYAADC01) in participants with moderately advanced Parkinson disease (PD) and motor fluctuations. Methods PD-1101 was a phase 1b, open-label, dose escalation trial of VY-AADC01, an e...

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Bibliographic Details
Main Authors: Bankiewicz, K.S (Author), Christine, C.W (Author), Fine, E.M (Author), Khwaja, O.S (Author), Larson, P.S (Author), Li, C. (Author), Liang, G.S (Author), Meier, A. (Author), Pfau, M.L (Author), Richardson, R.M (Author), Roberts, E.W (Author), Rodman, J.R (Author), Thompson, M.E (Author), van Laar, A.D (Author)
Format: Article
Language:English
Published: Lippincott Williams and Wilkins 2022
Subjects:
adult
aromatic levo amino acid decarboxylase
Article
atrial fibrillation
clinical article
Clinical Global Impression scale
cohort analysis
controlled study
enzyme activity
female
gene therapy
human
intraoperative period
lung embolism
male
middle aged
motor dysfunction
motor performance
nuclear magnetic resonance imaging
Parkinson disease
patient participation
patient safety
phase 1 clinical trial
practice guideline
quality of life
small intestine obstruction
Unified Huntington Disease Rating Scale
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