Table 2.
TEAEs, events, and compliance rates at 90 days or until discontinuation.
| TEAE | N = 20 |
|---|---|
| Any TEAEs, n (%) | 6 (30) |
| Elevated creatinine kinase levels, n (%) | 3 (15) |
| Liver function test abnormalities, n (%) | 1 (5) |
| Glucose tolerance abnormalities, n (%) | 1 (5) |
| Hyperammonemia, n (%) | 1 (5) |
| Anorexia, n (%) | 1 (5) |
| Dysgeusia, n (%) | 1 (5) |
| Vasculitis, n (%) | 1 (5) |
| Event | |
| Pneumonia, n (%) | 0 |
| PEG, n (%) | 0 |
| NPPV, n (%) | 1 (5) |
| TPPV, n (%) | 0 |
| Death, n (%) | 0 |
| Medication intake | |
| Number of missed medication doses | 1 (0, 43) |
| Adherence rate (%) | 99.7 (76.1, 100) |
TEAE, treatment-emergent adverse event; PEG, percutaneous endoscopic gastrostomy; NPPV, noninvasive positive pressure ventilation; TPPV, tracheostomy positive pressure ventilation. Data are expressed as the mean ± standard deviation or median (minimum, maximum) for continuous variables and as frequencies and percentages for discrete variables.