Table 2.
TEAEs of special interest in children with three copies of SMN2
| Category of AESI | n = 15a |
|---|---|
| Preferred term | n (%) |
| Hepatotoxicity | |
| Any TEAE | 4 (27) |
| Aspartate aminotransferase increased | 4 (27) |
| Alanine aminotransferase increased | 3 (20) |
| Blood alkaline phosphatase increased | 1 (7) |
| Gamma-glutamyltransferase increased | 1 (7) |
| Thrombocytopenia | |
| Any TEAE | 2 (13) |
| Hematemesis | 1 (7) |
| Hematochezia | 1 (7) |
| Contusion | 1 (7) |
| Cardiac adverse events | |
| Any TEAE | 3 (20) |
| Blood creatine phosphokinase MB increased | 2 (13) |
| Troponin increased | 2 (13) |
| Thrombotic microangiopathy | |
| Any TEAE | 0 (0) |
| Thrombocytopenia | 0 (0) |
| Sensory abnormalities suggestive of dorsal root ganglionopathy | |
| Any TEAE | 1 (7) |
| Areflexia | 1 (7) |
aSafety population: n = 6 males and n = 9 females; mean (s.d.) age at dosing, 28.7 (11.68) days.