Table 1.
Demographics and baseline characteristics of patients aged 5 years or older in the STRIDE Registry evaluable population
| All patients | |
|---|---|
| N = 268 | |
| Baselinea weight, kg | |
| n | 226 |
| Mean (SD) | 31.7 (14.1) |
| 95% CI | 29.8, 33.6 |
| Median | 27.5 |
| Min, max | 13.5, 87.0 |
| Baselinea height, cm | |
| n | 191 |
| Mean (SD) | 123.9 (15.8) |
| 95% CI | 121.6, 126.1 |
| Median | 121.1 |
| Min, max | 94.3, 178.0 |
| Baselinea BMI, kg/m2 | |
| n | 191 |
| Mean (SD) | 19.4 (4.5) |
| 95% CI | 18.7, 20.0 |
| Median | 18.2 |
| Min, max | 13.0, 40.1 |
| Age at first symptoms, years | |
| n | 253 |
| Mean (SD) | 2.9 (1.7) |
| 95% CI | 2.7, 3.1 |
| Median | 3.0 |
| Min, max | 0.1, 8.0 |
| Age at muscle biopsy, years | |
| n | 155 |
| Mean (SD) | 4.6 (2.4) |
| 95% CI | 4.2, 5.0 |
| Median | 4.3 |
| Min, max | 0.4, 13.0 |
| Age at genetic confirmation of nmDMD diagnosis, years | |
| n | 259 |
| Mean (SD) | 5.4 (3.1) |
| 95% CI | 5.0, 5.8 |
| Median | 5.0 |
| Min, max | 0.0b, 23.0 |
| Previously enrolled in ataluren clinical trial, n (%) | |
| No | 197 (73.5) |
| Yes | 71 (26.5) |
| Age at informed consent, years | |
| n | 268 |
| Mean (SD) | 10.7 (4.0) |
| 95% CI | 10.3, 11.2 |
| Median | 10.2 |
| Min, max | 3.7, 28.3 |
| Age at first visit captured within the registry, years | |
| n | 268 |
| Mean (SD) | 10.8 (4.0) |
| 95% CI | 10.3, 11.3 |
| Median | 10.2 |
| Min, max | 3.7, 28.3 |
| Age at cutoff date, years | |
| n | 268 |
| Mean (SD) | 14.8 (3.9) |
| 95% CI | 14.3, 15.3 |
| Median | 14.6 |
| Min, max | 6.1, 29.1 |
| Age at ataluren start date, years | |
| n | 268 |
| Mean (SD) | 9.8 (3.9) |
| 95% CI | 9.4, 10.3 |
| Median | 8.9 |
| Min, max | 5.0, 24.2 |
| Total ataluren use duration, days | |
| n | 268 |
| Mean (SD) | 1671 (566.8) |
| 95% CI | 1603, 1739 |
| Median | 1713 |
| Min, max | 133, 2736 |
BMI body mass index, CI confidence interval, nmDMD nonsense mutation Duchenne muscular dystrophy, SD standard deviation, STRIDE Strategic Targeting of Registries and International Database of Excellence
aBaseline data are data collected at first visit captured in the STRIDE Registry
bA minimum age value of zero was recorded owing to a prenatal diagnosis. Patients with a prenatal diagnosis recorded as the first symptom were excluded from the effectiveness analyses but were included in the evaluable population