Table 1.
Baseline Characteristics of the Study Participants
| Patient 101 | Patient 102 | Patient 103 | Patient 201 | Patient 202 | Patient 203 | Patient 204 | |
|---|---|---|---|---|---|---|---|
| Sex | M | M | M | M | F | M | F |
| Age at diagnosis (months) | 6.5 | 15 | 17 | 18 | 0 | 3.5 | 6 |
| Age ERT started (months) | 8 | 15 | 18 | 108 | 96 | 4.5 | 6 |
| Age-invasive ventilation started (months) | 42 | 7 | 29 | 28 | 96 | 20 | 18 |
| Age at enrollment (months) | 96 | 108 | 66 | 180 | 179 | 75 | 30 |
| MIP, cm H2O (% predicted) | −25.1 (26) | −1.6 (2) | −6.4 (8) | −2.6 (3) | N/A | −4.3 (5) | −35.5 (40) |
| MVV, liter/min (% predicted) | 7.0 (14.9) | 1.0 (2.1) | 2.1 (6.9) | 0.5 (0.8) | N/A | 1.6 (7.0) | 2.1 (12.7) |
| Off-ventilator tolerance (min/day) | 8 | 0.5 | 1.1 | 0.4 | N/A | 0.9 | 1.5 |
| Initial weight (kg) | 27 | 35 | 18.5 | 47 | 29 | 24.5 | 13 |
M, male; F, female; ERT, enzyme replacement therapy; MIP, maximal inspiratory pressure; MVV, maximal voluntary ventilation; N/A, not available.