Table 1.
Demographic characteristics of the analysis populations in FACHILD and the comparative FACOMS cohort. Numbers after the scales indicate maximum possible score.
| FACHILD | FACOMS | |
|---|---|---|
| N | 89 | 298 |
| Sex (male, %) | 41 (46.1%) | 163 (54.7%) |
| Age | 13.3 (2.8) | 13.2 (3.0) |
| Age of onset | 7.0 [5.0, 10.0] | 8.0 [5.0, 11.0] |
| GAA1 | 766.0 [699.0, 893.8] | 750.0 [650.0, 856.0] |
| GAA2 | 1000.0 [927.0, 1129.8] | 950 [833.0, 1057.0] |
| Compound Heterozygotes | 8 (9.0%) | 20 (6.7%) |
| mFARS total (93) | 37.8 (11.7) | 40.6 (11.0) |
| Upright stability (FARS E, 36) | 22.3 (5.3) | 21.7 (5.6) |
| Upper limbs (FARS B, 36) | 9.2 (5.3) | 12.0 (5.0) |
| Lower limbs (FARS C, 16) | 5.9 (2.4) | 6.4 (2.8) |
| Bulbar function (FARS A, 5) | 0.3 (0.5) | 0.6 (0.6) |
Data are n (%), median [IQR] or mean (SD).