Table 4 Exploratory outcomes of change from baseline in functional outcomes
Cohort 1 (dose, 1.85 × 1013 vg kg−1)a (n = 3) | Cohort 2 (dose, 7.41 × 1013 vg kg−1)b (n = 3) | Natural history cohort (n = 5) | |||||||
|---|---|---|---|---|---|---|---|---|---|
Month 6 (n = 3) | Year 1 (n = 3) | Year 2 (n = 3) | Month 6 (n = 3) | Year 1 (n = 2) | Year 2 (n = 2) | Month 6 (n = 5) | Year 1 (n = 5) | Year 2 (n = 5) | |
NSAD score | |||||||||
Mean (s.d.) | 3.0 (1.7) | 5.7 (1.5) | 5.7 (1.5) | 3.7 (3.5) | 4.0 (1.4) | 0.5 (0.7) | –2.2 (3.7) | –2.0 (2.1) | –5.3 (3.1) |
Median (range) | 4.0 (1.0, 4.0) | 6.0 (4.0, 7.0) | 6.0 (4.0, 7.0) | 4.0 (0, 7.0) | 4.0 (3.0, 5.0) | 0.5 (0, 1.0) | −1.0 (−7.0, 2.0) | −2.0 (−4.0, 1.0) | −6.0 (−8.0, −2.0) |
Time to rise, s | – | – | – | ||||||
Mean (s.d.) | −0.2 (0.8) | −0.8 (0.4) | −0.6 (0.2) | −1.3 (0.9) | −1.1 (1.1) | −0.7 (0.4) | |||
Median (range) | −0.6 (−0.7, 0.8) | −0.6 (−1.2, −0.5) | −0.6 (−0.7, −0.4) | −0.8 (−2.3, −0.7) | −1.1 (−1.8, −0.3) | −0.7 (−1.0, −0.4) | |||
Four-stair climb, s | – | – | – | ||||||
Mean (s.d.) | −0.5 (0.4) | −0.5 (0.3) | −0.3 (0.4) | −0.4 (0.3) | −0.4 (0) | −0.3 (0.3) | |||
Median (range) | −0.4 (−0.9, −0.1) | −0.5 (−0.8, −0.2) | −0.5 (−0.6, 0.2) | −0.4 (−0.7, −0.1) | −0.4 (−0.4, −0.4) | −0.3 (−0.5, −0.1) | |||
100-m walk, s | |||||||||
Mean (s.d.) | −3.8 (2.9) | −5.3 (3.2) | −2.8 (6.4) | −6.3 (6.7) | −7.9 (5.4) | −2.9 (9.7) | −0.8 (5.6) | 1.6 (6.8) | 5.2 (10.2) |
Median (range) | −4.6 (−6.2, 0.6) | −3.6 (−8.9, −3.3) | −4.4 (−8.2, 4.2) | −2.8 (−14.0, −2.1) | −7.9 (−11.7, −4.1) | −2.9 (−9.7, 4.0) | 1.2 (−7.5, 5.5) | 3.3 (−8.1, 7.7) | 9.0 (−6.4, 13.0) |
10-m walk, s | |||||||||
Mean (s.d.) | −0.6 (0.3) | −0.6 (0.2) | −0.2 (0.5) | −0.6 (0.6) | −0.6 (0.2) | −0.3 (0.9) | −0.7 (0.6) | −0.2 (0.7) | −0.1 (0.3) |
Median (range) | −0.6 (−0.9, −0.3) | −0.5 (−0.9, −0.5) | −0.3 (−0.6, 0.3) | −0.3 (−1.3, −0.2) | −0.6 (−0.7, −0.4) | −0.3 (−0.9, 0.4) | −0.7 (−1.5, 0) | −0.1 (−1.0, 0.6) | −0.1 (−0.3, 0.1) |
