Table 1.
Skeletal and respiratory muscle involvement.
| N (%) | Mean ± SD (median, range) | |
|---|---|---|
| All participants | 225 (100) | |
| Gait disturbance at the time of survey | 121 (53.8) | |
| Gait abnormality | 47 (20.8) | |
| With device | 13 (5.8) | |
| With wheelchair | 61 (27.1) | |
| Age at introduction (years) | 36.5 ± 15.8 (35, 9–72) | |
| Part‐time use | 15 (6.7) | |
| Full‐time use | 36 (16.0) |
| N | ||
|---|---|---|
| Serum CK | ||
| At initial survey | 212 | |
| Age (years) | 21.8 ± 16.9 (18, 0–73) | |
| Value (IU/L) | 3,582 ± 5,308 (1,480, 52–43,870) | |
| At final survey | 206 | |
| Age (years) | 30.7 ± 17.8*** (28, 0–81) | |
| Value (IU/L) | 1,783 ± 3,154*** (940, 22–30,850) | |
| Mean value between two points | 194 | |
| Age (years) | 26.4 ± 15.3 (25.5, 0–69) | |
| Value (IU/L) | 2,690 ± 3,445 (1,480, 118–30,529) | |
| %FVC | ||
| At initial survey | 132 | |
| Age (years) | 28.7 ± 16.1 (28, 4–73) | |
| Value (%) | 91.5 ± 17.7 (91.5, 30.4–132.9) | |
| At final survey | 150 | |
| Age (years) | 34.8 ± 16.6** (34, 5–81) | |
| Value (%) | 90.6 ± 19.5* (91.2, 15.9–133.9) | |
| Mean value between two points | 124 | |
| Age (years) | 32.7 ± 16.3 (33, 4.5–77) | |
| Value (IU/L) | 91.6 ± 16.9 (92.3, 23.2–131.2) |
| N (%) | ||
|---|---|---|
| Use of artificial ventilator | 15 (6.7) | |
| Night‐time only | 7 (3.1) | |
| Intermittent (daytime) | 1 (0.4) | |
| All day | 6 (2.7) | |
| NPPV introduction | 15 (6.7) | |
| Age at introduction | 36.6 ± 14.2 (34, 18–61) | |
| TPPV introduction | 4 (1.8) | |
| Age at introduction | 39.5 ± 13.7 (34.5, 30–59) |
*p < 0.05; **p < 0.01; ***p < 0.001 versus mean value at initial survey; ns, not significant versus mean value at initial survey.
CK, creatine kinase; FVC, forced vital capacity; IU, international units; NPPV, non‐invasive positive pressure ventilation; SD, standard deviation; TPPV, tracheostomy positive pressure ventilation.