Table 2.
Baseline median protein levels in whole blood
| Category | Baseline median SMN protein (ng/mL) | Min–max (ng/mL) |
|---|---|---|
| SMA type | ||
| Type 1 | 2.02 | 0.76–3.45 |
| Type 2 | 3.30 | 0.53–6.39 |
| Type 3 | 4.02 | 1.01–8.16 |
| Copy number | ||
| 2 copies | 1.76 | 1.21–3.45 |
| 3 copies | 3.25 | 0.53–6.91 |
| 4 copies | 4.69 | 2.16–8.16 |
| Previous treatment | ||
| Onasemnogene abeparvovec | 2.86 | 0.53–5.53 |
| MOONFISH study | 3.55 | 2.16–8.16 |
| Nusinersen | 3.09 | 1.01–6.91 |
| Olesoxime | 3.50 | 1.43–6.39 |
| Age quartile | ||
| ≤ 10.4 years | 3.06 | 0.53–5.53 |
| > 10.4 years–15 years | 3.26 | 1.21–6.39 |
| > 15 years–22.9 years | 3.56 | 1.01–6.68 |
| > 22.9 years | 3.55 | 1.36–8.16 |
| Weight quartile | ||
| ≤ 26 kg | 2.86 | 0.53–6.39 |
| > 26 kg–39 kg | 3.14 | 1.01–5.98 |
| > 39 kg–55.3 kg | 3.45 | 1.58–6.02 |
| > 55.3 kg | 3.84 | 1.36–8.16 |
SMA spinal muscular atrophy, SMN survival of motor neuron