Table 1.
Subject characteristics collected at the initiation of each study
| UX023-CL002 | UX023-CL201 | UX023-CL205 | UX023-CL301 | ||
|---|---|---|---|---|---|
| Assessment | (N = 103) | (N = 52) | (N = 13) | (N = 61) | Total |
| Age of historical data, years, min–max | 1–14 | 0–12 | 0–4 | 0–12 | 0–14 |
| Number of subjects included from the study, n | 103 | 52 | 13 | 60a | 228 |
| Female, n (%) | 67 (65) | 28 (54) | 4 (31) | 33 (55) | 132 (58) |
| White, n (%) | 71 (69) | 46 (89) | 12 (92) | 49 (82) | 178 (78) |
| Number of subjects who received prior supplementation therapy, n (%) | 102 (99) | 50 (96) | 13 (100) | 60 (100) | 225 (99) |
| Age at initiation of supplementation therapy, years, mean (SD) | 2.5 (2.2) | 2.1 (1.3) | 1.6 (1.5) | 2.3 (2.3) | 2.3 (2.1) |
| Birthday range | 1970–2012 | 2002–2010 | 2011–2015 | 2004–2016 | 1970–2016 |
| RSS total score, mean (SD)b | 1.2 (0.88)c | 1.8 (1.09) | 2.9 (1.37) | 3.2 (1.06) | 2.3 (1.34) |
Abbreviations: max, maximum; min, minimum; SD, standard deviation.
a One subject lacked prestudy growth data.
b Baseline data were used for CL201, CL205, and CL301. For CL002, the last measurement collected from the patient’s medical record was used.
c Only the subgroup of patients with radiographs was included, n = 60.