1. Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial 
Yin-Hsiu Chien, MD, Ni-Chung Lee, MD, Sheng-Hong Tseng, MD, Chun-Hwei Tai, MD, Prof Shin-ichi Muramatsu, MD, Prof Barry J Byrne, MD, Prof Wuh-Liang Hwu, MD 
The Lancet Child & Adolescent Health 
Volume 1, Issue 4, Pages (December 2017)
DOI: /S (17)
Copyright © 2017 Elsevier Ltd Terms and Conditions

2. Figure 1
Trial design
After enrolment, we did baseline assessments over the course of 30 days. Patients were assessed at days 3–7 after surgery to monitor for surgical complications and at 12 months to evaluate efficacy and other safety parameters. AAV2-hAADC=adeno-associated virus serotype 2 vector encoding human aromatic l-amino acid decarboxylase.
The Lancet Child & Adolescent Health 2017 1, DOI: ( /S (17) )
Copyright © 2017 Elsevier Ltd Terms and Conditions

3. Figure 2 Improvements in developmental scales after gene therapy
Data are mean scores, and error bars are standard deviation. PDMS-2=Peabody Developmental Motor Scales (second edition). AIMS=Alberta Infant Motor Scale. Bayley-III=Bayley Scale of Infant and Toddler Development (third edition).
The Lancet Child & Adolescent Health 2017 1, DOI: ( /S (17) )
Copyright © 2017 Elsevier Ltd Terms and Conditions

4. Figure 3 Bodyweight and AIMS raw scores
AIMS was done only after enrolment; therefore, the first datapoint of AIMS indicates the time of gene therapy. AIMS=Alberta Infant Motor Scale.
The Lancet Child & Adolescent Health 2017 1, DOI: ( /S (17) )
Copyright © 2017 Elsevier Ltd Terms and Conditions

5. Figure 4 FDOPA-PET images of patient 2
FDOPA uptake in the bilateral putamen was barely visible at baseline (0 months) but was clearly visible (arrows) after gene therapy (12 months). See appendix for PET images of other patients. FDOPA=6-[18F]-fluoro-l-DOPA.
The Lancet Child & Adolescent Health 2017 1, DOI: ( /S (17) )
Copyright © 2017 Elsevier Ltd Terms and Conditions