Table 1.
Demographic and Baseline Characteristics
| Edasalonexent 100 mg/kg (N = 88) | Placebo (N = 43) | Total (N = 131) | |
| Age at Screening (Year), Mean (SD) | 5.65 (1.048) | 5.77 (0.995) | 5.69 (1.029) |
| Age Group, n (%)a | |||
| ≤6.0 yr. | 55 (62.5) | 29 (67.4) | 84 (64.1) |
| > 6.0 yr. | 33 (37.5) | 14 (32.6) | 47 (35.9) |
| Age at Diagnosis | 3.82 (1.70) | 3.59 (1.94) | 3.75 (1.78) |
| ≤6.0 yr. | 3.11 (1.50) | 3.43 (1.67) | 3.22 (1.56) |
| > 6.0 yr. | 4.96 (1.37) | 3.93 (2.48) | 4.68 (1.78) |
| Age at Symptom Onset | 2.90 (1.77) | 2.65 (1.79) | 2.82 (1.77) |
| ≤6.0 yr. | 2.41 (1.37) | 2.78 (1.69) | 2.54 (1.49) |
| > 6.0 yr. | 3.67 (2.06) | 2.39 (2.01) | 3.30 (2.11) |
| Race, n (%) | |||
| White | 74 (84.1) | 38 (88.4) | 112 (85.5) |
| Black or African American | 4 (4.5) | 1 (2.3) | 5 (3.8) |
| Asian | 3 (3.4) | 2 (4.7) | 5 (3.8) |
| Multiracial | 3 (3.4) | 1 (2.3) | 4 (3.1) |
| Native Hawaiian/Other Pacific Islander | 1 (1.1) | 0 (0.0) | 1 (0.8) |
| Other | 2 (2.3) | 1 (2.3) | 3 (2.3) |
| Unknown | 1 (1.1) | 0 | 1 (0.8) |
| Ethnicity, n (%) | |||
| Hispanic or Latino | 14 (15.9) | 6 (14.0) | 20 (15.3) |
| Not Hispanic or Latino | 69 (78.4) | 35 (81.4) | 104 (79.4) |
| Not Reported/unknown | 5 (5.7) | 2 (4.7) | 7 (5.3) |
| Region, n (%) | |||
| North America | 63 (71.6) | 31 (72.1) | 94 (71.8) |
| Europe/Asia/Australia | 25 (28.4) | 12 (27.9) | 37 (28.2) |
| Treatment with Eteplirsen, n (%) | |||
| Yes | 2 (2.3) | 2 (4.7) | 4 (3.1) |
| No | 86 (97.7) | 41 (95.3) | 127 (96.9) |
| Time to Stand from Supine, n (%) | |||
| ≤5 seconds | 48 (55.8) | 26 (60.5) | 74 (57.4) |
| > 5 seconds | 38 (44.2) | 17 (39.5) | 55 (42.6) |
| Missing, n (%) | 2 (2.3) | 0 | 2 (1.6) |
| Previous corticosteroid use | 2 (2.3) | 2 (4.7) | 4 (3.1) |
| Mutation type | |||
| Deletion | 66 (76.1) | 31 (72.1) | 97 (74.1) |
| Duplication | 18 (19.3) | 8 (18.6) | 26 (19.1) |
| Nonsense | 4 (4.5) | 4 (9.3) | 8 (6.1) |
aPatient stratification by age included those from 4 years of age through ≤6.0 yr. and those > 6.0 yr.