Table 1.
Patient characteristics at time of gene therapy. To reduce the potential risk of re-identification of individual patients, we provide ranges for age and body weight and do not disclose the sex of individual patients in this table. *Values refer to time of gene therapy
| Patient No. | Weight range* (kilogram) | Age range* (months) | Functional status* | Respiratory support and tube feeding* | Age at disease onset (months) | Previous drug treatment (age at start in months) | Best motor function before treatment with nusinersen | SMN2 copy number |
| #1 | 7–7.9 | 19–21 | Sitting without support | None | 4–6 | Nusinersen (13–15) | Sitting without support | 3 |
| #2 | 8–8.9 | 10–12 | Sitting without support | None | 4–6 | Nusinersen (7–9) | Head control | 2 |
| #3 | 9–9.9 | 16–18 | Sitting without support | None | 7–9 | Nusinersen (13–15) | Sitting without support | 3 |
| #4 | 9–9.9 | 10–12 | No head control | Nocturnal non-invasive ventilation, tube for additional fluids | 1–3 | Nusinersen (1–3) | No head control | 2 |
| #5 | 10–10.9 | 16–18 | Sitting without support | None | 1–3 | Nusinersen (7–9) | Head control | 3 |
| #6 | 10–10.9 | 37–39 | Sitting without support | None | 10–12 | Nusinersen (28–30) | Sitting without support | 3 |
| #7 | 11–11.9 | 19–21 | Walking | None | Newborn-screening | Nusinersen (1–3) | n.a. | 2 |
| #8 | 10–10.9 | 10–12 | Sitting without support | None | Newborn-screening | Nusinersen (1–3) | n.a. | 3 |