Table 1.

Patient demographics of SMA patients.

Case number	SMA type	SMN2 copy number	Gender	Age at diagnosis	Family history	Consanguinity	Prior therapy (number of doses) [age at start]	Ventilation	Age at infusion	Gene dosing weight (kg)	Temperature at infusion (°C)	O2 Saturation at infusion (%)	HR at infusion (beat/min)	RR at infusion (breaths/min)	BP at infusion (mm Hg)
1	1	2	Male	3 m	Yes	Yes	Nusinersen (7) [100 d]	SIMV	1 y 11 m	7.7	36.5	100	100	24	100/60
2	1	2	Male	4 d	Yes	Yes	Nusinersen (7) [10 d]	None	1 y 6 m	7.8	36.5	98	126	28	95/49
3	1	2	Male	37 d	Yes	Yes	Nusinersen (7) [41 d]	BiPAP	1 y 5 m	4.5	36.6	99	115	34	95/41
4	1	2	Male	6 m	No	No	Nusinersen (6) [195 d]	None	1 y 7 m	10.2	36.6	100	96	24	95/50
5	1	2	Male	3 m	Yes	Yes	Nusinersen (4) [102 d]	None	1 y 0 m	8.7	36.8	96	130	40	86/50
6	2	3	Female	21 m	No	No	None	None	1 y 11 m	10.1	36	98	108	23	77/57
7	1	2	Male	2 m	Yes	Yes	Nusinersen (3) [96 d]	None	0 y 4 m	6.0	37.3	100	118	36	101/67
8	1	2	Male	2 m	Yes	Yes	Nusinersen (5) [71 d]	None	0 y 8 m	6.9	36.5	100	120	34	92/62
9	2	3	Female	18 m	Yes	Yes	None	None	1 y 9 m	10.5	36.4	100	121	24	94/52

BiPAP bilevel positive airway pressure, BP blood pressure, d days, m months, HR heart rate, RR respiration rate, SIMV synchronized intermittent mandatory ventilation, SMA spinal muscular atrophy, y year.