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Table 3 Clinical characteristics for SMA patients (N = 101)

From: Treatment preference among patients with spinal muscular atrophy (SMA): a discrete choice experiment

  Patient (n = 65) Patient represented by caregiver (n = 36)
Age range when first diagnosed with SMA 6 mos. to 59 yrs prenatal to 26 yrs
Age when first symptoms associated with SMA, n (%)   
 < 6 months old 13 (20.0) 18 (50.0)
 6–18 months old 30 (46.2) 11 (30.5)
 18 months—3 years old 13 (20.0) 03 (08.3)
 4–18 years old 07 (10.8) 01 (02.7)
 > 18 years old 02 (03.1) 02 (05.5)
Type of SMA, n (%)   
 Type 1 04 (6.15) 17 (47.22)
 Type 2 33 (50.77) 15 (41.67)
 Type 3 27 (41.54) 02 (05.55)
 Type 4 01 (1.54) 0
 Unknown 0 02 (05.55)
Self-reported level of physical (motor) functioning or ability, n (%)   
 Walking alone 8 (12.31) 1 (2.77)
 Sitting without support 23 (35.38) 10 (27.78)
 Walking with assistance 7 (10.77) 3 (8.33)
 Standing with assistance 1 (1.54) 1 (2.77)
 Hands and knee crawling 0 2 (5.55)
 No motor function 3 (4.62) 3 (8.33)
 None of the above; have some motor function 23 (35.38) 16 (44.44)
Self-reported breathing ability, n (%)   
 Can breathe without mechanical support 55 (84.61) 29 (80.55)
 Need mechanical support to breathe for some of the day 7 (10.77) 3 (8.33)
 Need mechanical support to breathe for most of the day 1 (1.54) 0
 Need 24-h mechanical support to breathe 2 (3.08) 4 (11.11)
Other illness limits daily activities, n (%)   
 Yes 10 (15.38) 2 (5.55)
Current Treatment, n (%)a   
 Supportive care only (not currently using a medical treatment for SMA) 14 (21.54) 6 (16.67)
 Spinraza® (Nusinersen) 41 (63.08) 18 (50.00)
 Zolgensma® (Onasemnogene Abeparvovec-xioi) 0 10 (27.78) a
 Other 1 (1.54) 0
 None 9 (13.84) 3 (11.11)
Surgeries before, n (%)a   
 Spinal fusion 32 (49.23) 7 (19.44)
 Spinal rods 24 (36.92) 8 (22.22)
 Gastrostomy (G-tube) 9 (13.84) 11 (30.56)
 Joint contracture 4 (6.15) 0
 Tracheotomy 6 (9.23) 8 (22.22)
 None 26 (40.00) 17 (47.22)
Current tools/equipment use, n (%)a   
 Breathing machine/mechanical ventilation 15 (23.1) 11 (30.56)
 Feeding tube 5 (7.7) 10 (27.78)
 Suction machine to help clear throat 13 (20.0) 16 (44.44)
 Cough assist machine 22 (33.8) 24 (66.67)
 Walking frame 2 (3.1) 2 (5.55)
 Wheelchair 53 (81.5) 20 (55.56)
 Other 13 (20.0) 9 (25.00)
 None 6 (9.23) 6 (16.67)
  1. Mos., months; Yrs., years
  2. aAnswer options are not mutually exclusive of one another