Fig. 3: Changes in Oculogyric Crises (OGC) and motor function after gene delivery.

A The OGC score was calculated monthly, and represents the weekly average of the duration of episodes (hours per week), weighted by severity (grade 1–3: 1 = mild/eye deviation only; 2 = moderate/eye deviation + dystonia or dyskinesia of the face and/or neck; 3 = severe/dystonia or dyskinesia involving the trunk and/or limbs). By Month 3, OGCs completely resolved in 6/7 subjects. Inset (black arrow): Subject 1 had residual episodes throughout the 24-month study period; the severity decreased after surgery (red arrow). B Gross Motor Function Measure-88 (GMFM-88) scores for Cohorts 1 (red) and 2 (blue). Data are shown through Month 24 for Subjects 1–3, Month 18 for Subjects 4 and 5, and Month 6 for Subjects 6 and 7. Baseline GMFM scores for all subjects (circled) were ≤10, consistent with severe motor impairment. Inset: changes in GMFM score between Baseline and Month 12. An increase of ≥7 points (dotted line, representing a clinically meaningful positive change) was observed in 6/7 subjects by Month 12. Source data are provided as a Source Data file.