Table 4.
Coefficient | SE | 95% CI | p value | ||
---|---|---|---|---|---|
Level of change in motor function (ref: Stable) | |||||
Better | 0.65 | 0.09 | 0.47 | 0.83 | 0.000 |
Change in breathing function (ref: Stable) | |||||
Better | 0.79 | 0.10 | 0.60 | 0.98 | 0.000 |
Drug is approved for: (ref: Pediatric and adult patients with SMA) | |||||
Pediatric patients with SMA under 2 years of age | − 1.28 | 0.10 | − 1.47 | − 1.09 | 0.000 |
Route and frequency of treatments (ref: Intrathecal spinal injection—3–6 times per year) | |||||
Intravenous injection—one-time treatment | 0.51 | 0.11 | 0.30 | 0.73 | 0.000 |
Oral—daily medication | 0.80 | 0.13 | 0.55 | 1.04 | 0.000 |
Treatments potential risk (ref: Mild risk) | |||||
Moderate risk | − 0.76 | 0.13 | − 1.01 | − 0.50 | 0.000 |
Serious, or life-threatening risk | − 1.86 | 0.13 | − 2.11 | − 1.60 | 0.000 |
SE, standard error; CI, confidence interval