Table 2.
MFM results for nusinersen treated and untreated cohorts
| Source | SMA TYPE | MFM baseline (mean + SD or mean (range)) | Time from infusion/FU timing | n | Mean change mean (SD) | Age at baseline (range) |
|---|---|---|---|---|---|---|
| Nusinersen-treated cohorts | ||||||
| Audic 2020 | sma 1–2 | 45 (10–87) | T12 | 33 | 7 (SD not reported) | 2–5 |
| Gomez Garcia 2020 | sma 1–2 | 25 + 12 | T14 | 3 | 5 (SD not reported) | 3.5–4.7 |
| Audic 2020 | sma 1–2 | 40 (4–60) | T12 | 35 | 3 (SD not reported) | 6–17 |
| Gomez Garcia 2020 | sma 1–2 | 37 + 17 | T14 | 13 | 9 (SD not reported) | 6.8–11.5 |
| Gomez Garcia 2020 | sma 1–2 | 34 + 17 | T14 | 16 | 9 (SD not reported) | 3.5–11.5 |
| Audic 2020 | sma 1–2 | 42 (4–87) | T12 | 68 | 5 (SD not reported) | 2–17 |
| Untreated cohorts | ||||||
| Annoussamy | SMA 2 NON SITTER | not reported | T12 | 14 | − 1.12 (2.29) | 2–30 |
| Annoussamy | SMA 2 NON SITTER | not reported | T24 | 11 | − 3.03 (3.77) | 2–30 |
| Annoussamy | SMA 2 SITTER | not reported | T12 | 11 | − 2.39 (5.18) | 2–30 |
| Annoussamy | SMA 2 SITTER | not reported | T24 | 4 | − 4.95 (8.69) | 2–30 |
| Annoussamy | SMA 3 NON AMBULANT | not reported | T12 | 7 | 0.35 (3.27) | 2–30 |
| Annoussamy | SMA 3 NON AMBULANT | not reported | T24 | 5 | − 0.83 (2.14) | 2–30 |
| Annoussamy | SMA 3 AMBULANT | not reported | T12 | 11 | − 1.67 (3.87) | 2–30 |