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Table 5 Treatment initiation, dosage and treatment duration in the efficacy population

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

  Isovaleric aciduria (n = 4) Methylmalonic aciduria (n = 25) Propionic aciduria (n = 19) Neonates(n = 29) Non-neonates(n = 19) Total(n = 48)
Time to treatment initiation
 Mean, days (SD) 0.8 (1.0) 2.5 (5.5) 4.2 (9.5) 1.5 (2.8) 5.3 (10.6) 3.0 (7.2)
 Median (range) 0.5 (0–2) 0.0 (0–24) 2.0 (0–41) 1.0 (0–14) 1.0 (0–41) 1.0 (0–41)
First dose
 Mean, mg/kg (SD) 99.0 (63.3) 78.2 (64.9) 119.5 (83.2) 106.7 (75.3) 80.4 (70.5) 96.3 (73.8)
 Median, mg/kg (range) 81.6 (50.0–182.7) 51.0 (13.7–236.2) 106.0 (13.3–303.0) 100.0 (13.7–303.0) 50.0 (13.3–256.4) 75.5 (13.3–303.0)
Initial 24-h dose
 Mean, mg/kg (SD) 216.0 (217.9) 160.6 (108.3) 209.3 (186.9) 214.1 (179.1) 139.2 (81.7) 184.5 (151.8)
 Median, mg/kg (range) 131.6 (62.5–538.5) 129.0 (41.1–470.8) 190.2 (53.1–909.1) 170.2 (41.1–909.1) 100 (45.3–333.3) 156.4 (41.0–909.1)
Treatment duration
 Mean, days (SD) 3.5 (1.7) 6.1 (4.6) 5.2 (5.0) 4.9 (3.5) 6.5 (5.9) 5.5 (4.6)
 Median, days (range) 3.5 (2–5) 5.0 (1–15) 3.0 (1–15) 4.0 (1–15) 3.0 (1–15) 4.0 (1–15)
  1. SD, standard deviation