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Table 2 Cumulative incidence and 95% CI of patients achieving the MSD of the total PDMS-2 score and each motor milestone

From: 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

 

Month 0

Month 6

Month 12

Month 18

Month 24

Month 30

Month 36

Month 42

Month 48

Month 54

Month 60

Month 66

Month 72

Month 78

Month 84

40pts PDMS-2 change

0 (0, 0)

50

(28, 65)

75

(53, 87)

86

(65, 94)

86

(65, 94)

90

(68, 97)

90

(68, 97)

95

(70, 99)

100

(70, 99)

100

(70, 99)

100

(70, 99)

100

(70, 99)

100

(70, 99)

100

(70, 99)

100

(70, 99)

Head control

0 (0, 0)

17

(2.2, 29)

52

(30, 67)

71

(48, 83)

75

(52, 87)

75

(52, 87)

87

(65, 96)

87

(65, 96)

87

(65, 96)

87

(65, 96)

87

(65, 96)

87

(65, 96)

87

(65, 96)

87

(65, 96)

87

(65, 96)

Sitting unassisted

0 (0, 0)

3.3

(0, 9.5)

25

(7.0, 39)

40

(18, 56)

56

(32, 71)

68

(44, 82)

68

(44, 82)

72

(48, 85)

77

(52, 89)

82

(56, 93)

82

(56, 93)

82

(56, 93)

82

(56, 93)

82

(56, 93)

82

(56, 93)

Standing with support

0 (0, 0)

3.3

(0, 9.5)

6.8

(0, 15)

11

(0, 21)

27

(7.5, 42)

31

(10, 47)

36

(14, 52)

44

(20, 61)

44

(20, 61)

44

(20, 61)

49

(24, 66)

49

(24, 66)

58

(28, 75)

58

(28, 75)

58

(28, 75)

Walking with assistance

0 (0, 0)

0 (0, 0)

0 (0, 0)

0 (0, 0)

4.2

(0, 12)

8.7

(0, 20)

14

(0, 27)

14

(0, 27)

14

(0, 27)

24

(3.0, 41)

24

(3.0, 41)

24

(3.0, 41)

24

(3.0, 41)

24

(3.0, 41)

24

(3.0, 41)