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 (%) | Â | Â |
 < 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 (%) | Â | Â |
 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 (%) | Â | Â |
 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 (%) | Â | Â |
 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 (%) | Â | Â |
 Yes | 10 (15.38) | 2 (5.55) |
Current Treatment, n (%)a | Â | Â |
 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 | Â | Â |
 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 | Â | Â |
 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) |