Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and correlation with Bayley-III scores and motor milestones

Table 1 Results from the MSD analyses: Mean-difference approach

	MeanTotal PDMS-2 score	Difference¹	N²
Motor milestone: Mastery
None	45.0		165
Head control	91.4	46.4	38
Sitting unassisted	127.0	35.6	62
Standing with support	173.0	46.0	32
Walking with assistance	225.0	52.0	17
Mean difference: 45.0
Motor milestone: Emergent and Mastery
None	42.4		149
Head control	83.7	41.3	41
Sitting unassisted	117.0	33.3	69
Standing with support	170.0	53.0	38
Walking with assistance	225.0	55.0	17
Mean difference: 45.6

¹The difference between means of adjacent Motor milestones (full-head control vs. none, sitting unassisted vs. full-head control, standing with support vs. sitting unassisted, and walking with assistance vs. standing with support)
²N = number of observations

ISSN: 1750-1172