Table 1 Baseline demographics and characteristics

From: Intrathecal onasemnogene abeparvovec in treatment-naive patients with spinal muscular atrophy: a phase 3, randomized controlled trial

 

OAV101 IT

(n = 75)

Sham

(n = 51)

Overall

(N = 126)

Age at dosing, years

 Mean (s.d.)

5.89 (3.58)

5.87 (3.05)

5.88 (3.36)

 Range

2.1–16.6

2.4–14.2

2.1–16.6

Mean (range) age at SMA symptom onset, months

12.7 (6–31)

12.6 (6–33)

12.7 (6–33)

Sex, n (%)

 Male

34 (45.3)

28 (54.9)

62 (49.2)

 Female

41 (54.7)

23 (45.1)

64 (50.8)

SMN2 copy number, n (%)

 2

1 (1.3)

4 (7.8)

5 (4.0)

 3

73 (97.3)

46 (90.2)

119 (94.4)

 ≥4

1 (1.3)

1 (2.0)

2 (1.6)

HFMSE scorea

 Mean (s.d.)

17.97 (10.11)

18.17 (9.76)

 

 Range

1.0–41.0

2.0–42.5

 

RULM scoreb

 Mean (s.d.)

16.52 (6.74)

17.42 (6.35)

 

 Range

4.0–31.7

4.3–31.0

 
  1. aScores range from 0 to 66, with higher scores indicating better motor function.
  2. bScores range from 0 to 37, with higher scores indicating better function.
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