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Cohort characteristics at inclusion
| Characteristic | COL6-RD (n=23) | LAMA2-RD (n=21) | P Value |
|---|---|---|---|
| Age, y, median (IQR) | 9 (7.0–13.5) | 7 (5.0–9.0) | .03 |
| Sex, male, n (%) | 12 (52) | 10 (48) | >.99 |
| Ambulant, n (%) | 16 (70) | 4 (19) | .002 |
| MFM-32 raw scores, median (IQR) | |||
| MFM D1 | 16 (7.0–24.0) | 1 (1.0–4.0) | .006 |
| MFM D2 | 32 (29.5–34.0) | 29 (15.0–31.0) | .01 |
| MFM D3 | 20 (18.5–20.0) | 14 (13.0–16.0) | <.001 |
| MFM TS | 69 (59.5–77.0) | 44 (27.0–52.0) | .002 |
Abbreviation: IQR, interquartile range.
