Table 1.
Selected published reports of very early nusinersen initiation in SMA.
| Study | SMN2 copies | Symptomatic at first dose | Age at first dose | Diagnostic setting/baseline features | Follow-up/reported outcome |
|---|---|---|---|---|---|
| Unver et al. | 2 | No | 7 h | Prenatal diagnosis; absent DTRs | Normal development reported at 13 months |
| Unver et al. | 3 | No | 3 days | Prenatal diagnosis; no overt SMA manifestations | Normal development reported at almost 6 years |
| Nishino et al. | 2 | Yes | 55 days | Postnatal diagnosis; hypotonia, absent DTRs | Respiratory and feeding deterioration reported at 6 months |
| Present case | 2 | Yes | 5 days | Postnatal diagnosis; generalized hypotonia, markedly reduced spontaneous movement, weak cry, tongue fasciculations, absent DTRs | Four loading doses completed by day 68; CHOP-INTEND increased from 6 to 30; no procedure-related adverse events |
The table includes selected published individual-level neonatal cases of very early nusinersen initiation with sufficient clinical detail for direct comparison. DTRs, deep tendon reflexes; SMA, spinal muscular atrophy.