Table 1 Patient demographics of SMA patients.

From: Gene therapy for spinal muscular atrophy: the Qatari experience

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
  1. 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.