Table 2.
Change in plasma ammonia concentration from baseline to study endpoint in efficacy population
| Episodes n | Mean change in NH3 at endpoint (μmol/L) | Mean time to the endpoint, days | |
|---|---|---|---|
| Total | 48 | -292.2 | 4.6 |
| Isovaleric aciduria | 4 | -614.3 | 2.8 |
| Methylmalonic aciduria | 25 | -229.2 | 5.2 |
| Propionic aciduria | 19 | -307.3 | 4.2 |
| Neonates | 29 | -407.6 | 4.0 |
| Non-neonates | 19 | -116.1 | 5.6 |
| With concomitant ammonia scavengers | 15 | -366.0 | 3.9 |
| Without concomitant ammonia scavengers/haemofiltration | 27 | -200.2 | 5.0 |
| With haemofiltration (with or without ammonia scavengers) | 6 | -521.8 | 4.8 |
NH3, ammonia