Table 3.

Dosing information for carglumic acid treatment

Patient number	Weight at birth (kg)	Weight at initiation of carglumic acid (kg)a	Weight at 6 months’ post-initiation of carglumic acid (kg)b	Weight at study enrolment (kg)c	Age at initiation of carglumic acid	Dose at initiation of treatment (mg daily)d [dose frequency]	Number of dose changes	Reason(s) for dose changes	Last recorded dose (mg daily)d [dose frequency]
1	3.06	NR	8.75	9.32	2 days	75 [QID]	2	Dosing schedule (n = 2)	100 [QID]
2	2.78	NR	NR	30.0	7.88 years	400 [TID]	0	–	400 [TID]
3	3.21	10.9	12.1	17.0	3.04 years	600 [BID]	0	–	600 [BID]
4	2.92	2.47	7.35	12.0	6 days	150 [OD]	2	Weight (n = 4)	200 [BID]
5	3.00	3.00	8.12	15.0	3 days	150 [BID]	3	Ammonia control (n = 3)	700 [BID]
6	3.45	12.1	14.3	17.0	1.81 years	600 [BID]	1	NR	800 [BID]
7	3.4	3.2	6.785	12.7	20 days	150 [QID]	0	–	150 [QID]
8	3.3	9.3	13	20.0	8.5 months	150 [OD]	1	Dosing schedule	150 [OD]
9	1.9	21.3	21.5	44.3	6.75 years	100 [BID]	5	Lack of response (n = 3), NR (n = 1)	200 [BID + OD]
10	3.32	12.8	14.4	30.08	3.10 years	100 [BID]	7	Growth and weight gain (n = 3), lack of response (n = 2), increased during an acute episode (n = 2)	200 [QID]

BID twice daily, NR not reported, OD once daily, QID four times daily, TID three times daily

^aInitiation of carglumic acid under usual clinical practice

^bRefers to 6 months after carglumic acid treatment was initiated under usual clinical practice, prior to study enrolment

^cRefers to the date at which consent was received for the patient to be enrolled in the study, which was after carglumic acid treatment was initiated under usual clinical practice

^dUnless otherwise indicated