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Patient characteristics (evaluated at screening and baseline)
| Demographics | Placebo treatment arm (n = 57) |
|---|---|
| Age, years | |
| Mean (SD) | 8.3 (2.33) |
| Median | 8.0 |
| Range | 5-15 |
| Race, n (%) | |
| White | 54 (94.7) |
| Black | 0 (0.0) |
| Asian | 1 (1.8) |
| Hispanic | 1 (1.8) |
| Other | 1 (1.8) |
| Body height, cm | |
| Mean (SD) | 123 (11.8) |
| Median | 122 |
| Range | 104-163 |
| Body weight, kg | |
| Mean (SD) | 29 (9.1) |
| Median | 26 |
| Range | 16-55 |
| Stop codon type, n (%) | |
| UGA | 31 (54.4) |
| UAG | 12 (21.1) |
| UAA | 14 (24.6) |
