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Table 2 Change in plasma ammonia concentration from baseline to study endpoint in efficacy population

From: Carglumic acid enhances rapid ammonia detoxification in classical organic acidurias with a favourable risk-benefit profile: a retrospective observational study

 

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

  1. NH3, ammonia