Linearised (A) and raw (B) NSAA total score: interactions coefficients for skippable genotypes at 12 and 24 months
| (A) | Linearised NSAA 12 months | Linearised NSAA 12+24 months |
|---|---|---|
| ALL DMD (n=395) | −5.0 (−6.5 to −3.5) p<0.001 | −13.8 (−15.7 to −12.0) p<0.001 |
| Skip 44 (n=27) | 2.5 (−3.2 to 8.3; p=0.39) | 9.1 (2.3 to 15.9; p<0.001)** |
| Skip 45 (n=31) | 1.8 (−3.6 to 7.4; p=0.50) | −3.2 (−9.7 to 3.5; p=0.35) |
| Skip 46 (n=34) | 2.8 (−2.4 to 8.0; p=0.29) | 8.8 (2.6 to 15.1; p<0.01)* |
| Skip 50 (n=8) | 4.3 (−6.6 to 15.2; p=0.44) | 8.3–4.0 to 20.7; p=0.19) |
| Skip 51 (n=61) | −2.5 (−6.7 to 1.8; p=025) | −5.7 (−10.6 to −0.9; p=0.02)* |
| Skip 52 (n=9) | 1.0 (−6.7 to 8.8; p=0.79) | 9.2 (0.4 to 18.0; p=0.04)* |
| Skip 53 (n=41) | −6.5 (−11.2 to −1.7; p<0.05)* | −14.2 (−19.9 to −8.5; p<0.001)** |
| (B) | NSAA 12 months | NSAA 12+24 months |
|---|---|---|
| ALL DMD (n=395) | −2.1 (−2.7 to −1.5) p<0.001 | −5.8 (−6.5 to −5.1) p<0.001 |
| Skip 44 (n=27) | 1.3 (−0.9 to 3.5; p=0.25) | 3.9 (1.3 to 6.5; p<0.01)* |
| Skip 45 (n=31) | 0.3 (−1.8 to 2.5; p=0.75) | −0.6 (−3.2 to 1.9; p=0.6) |
| Skip 46 (n=34) | 1.3 (−0.6 to 3.3; p=0.19) | 3.5 (1.1 to 5.9; p<0.01)* |
| Skip 50 (n=8) | 1.9 (−2.2 to 6.1; p=0.37) | 3.0(−1.8 to 7.7; p=0.22) |
| Skip 51 (n=61) | −1.0 (−2.6 to 0.6; p=022) | −2.4 (−4.2 to −0.5; p=0.01)* |
| Skip 52 (n=9) | 0.4 (−2.6 to 3.3; p=0.80) | 3.0 (−0.4 to 6.4; p=0.08) |
| Skip 53 (n=41) | −2.0 (−3.8 to −0.1; p=0.04)* | −4.5 (−6.7 to −2.3; p<0.001)** |
*p<0.05; **p<0.001
The overall slope of decline is described for the overall DMD population at 12 and 24 months. Skippable genotypes are compared with all DMD, at 1 and 2 years. Interaction coefficients (p value) suggest that boys skippable by exons 44 and 46 decline at a slower rate over the course of 2 years, while boys skippable by exons 51 and 53 decline faster.
DMD, Duchenne muscular dystrophy; NSAA, NorthStar Ambulatory Assessment.