Table 2.
Characteristics of survey respondents
| Total sample | MFIS | MFI | FSS | PedsQL MF | SMA-HI | |
|---|---|---|---|---|---|---|
| Total,, n | 253 | 158 | 162 | 146 | 150 | 142 |
| Gender, n (%) | ||||||
| Female | 164 (64.82) | 102 (64.56) | 113 (69.75) | 95 (65.07) | 90 (60.00) | 91 (64.08) |
| Age, in years | ||||||
| Mean (SD) | 38.21 (13.62) | 37.89 (12.56) | 37.96 (14.14) | 38.65 (13.36) | 37.53 (13.80) | 39.09 (14.25) |
| Range | 18–79 | 18–78 | 18–79 | 18–79 | 18–78 | 18–79 |
| Race, n (%) | ||||||
| White | 209 (82.61) | 134 (84.81) | 133 (82.10) | 120 (82.19) | 121 (80.67) | 118 (83.10) |
| Income, n (%) | ||||||
| ≤ $40,000 | 76 (30.04) | 51 (32.28) | 46 (28.40) | 41 (28.08) | 44 (29.33) | 46 (32.39) |
| $41,000-$100,000 | 92 (36.36) | 55 (34.81) | 57 (35.19) | 58 (39.73) | 60 (40.00) | 45 (31.69) |
| ≥ $101,000 | 39 (15.42) | 22 (13.92) | 28 (17.28) | 22 (15.07) | 22 (14.67) | 23 (16.20) |
| Unknown | 46 (18.18) | 30 (18.99) | 31 (19.14) | 25 (17.12) | 24 (16.00) | 28 (19.72) |
| Education, n (%) | ||||||
| Associate degree or less | 95 (37.55) | 56 (35.44) | 61 (37.65) | 50 (34.25) | 64 (42.67) | 54 (38.03) |
| Bachelor's degree | 81 (32.02) | 51 (32.28) | 53 (32.72) | 53 (36.30) | 43 (28.67) | 43 (30.28) |
| Master's degree or higher | 75 (29.64) | 50 (31.65) | 46 (28.40) | 42 (28.77) | 42 (28.00) | 44 (30.99) |
| Unknown | 2 (0.79) | 1 (0.63) | 2 (1.23) | 1 (0.68) | 1 (0.67) | 1 (0.70) |
| Age at diagnosis, in years, n | 246 | 154 | 158 | 141 | 143 | 141 |
| Mean (SD) | 8.13 (12.86) | 7.60 (12.47) | 8.48 (13.75) | 8.09 (12.85) | 8.49 (12.94) | 8.04 (12.28) |
| Range | 0–77 | 0–71 | 0–77 | 0–77 | 0–71 | 0–77 |
| SMA type, n (%) | ||||||
| Type I | 15 (5.93) | 9 (5.70) | 13 (8.02) | 7 (4.79) | 9 (6.00) | 7 (4.93) |
| Type II | 113 (44.66) | 73 (46.20) | 69 (42.59) | 66 (45.21) | 73 (48.67) | 58 (40.85) |
| Type III | 112 (44.27) | 66 (41.77) | 72 (44.44) | 68 (46.58) | 61 (40.67) | 68 (47.89) |
| Other/unknowna | 13 (5.14) | 10 (6.33) | 8 (4.94) | 5 (3.42) | 7 (4.67) | 9 (6.34) |
| SMN2 copies, n (%) | ||||||
| ≤ 2 copies | 36 (14.23) | 23 (14.56) | 23 (14.20) | 20 (13.70) | 21 (14.00) | 20 (14.08) |
| 3 copies | 81 (32.02) | 53 (33.54) | 52 (32.10) | 47 (32.19) | 49 (32.67) | 42 (29.58) |
| 4 or more copies | 36 (14.23) | 16 (10.13) | 38 (17.28) | 24 (16.44) | 18 (12.00) | 22 (15.49) |
| Unknown | 100 (39.53) | 66 (41.77) | 59 (36.42) | 55 (37.67) | 62 (41.33) | 58 (40.85) |
| Ever treated with an SMA-DMTb, n (%) | ||||||
| Yes | 191 (75.49) | 116 (73.42) | 122 (75.31) | 112 (76.71) | 114 (76.00) | 108 (76.06) |
| No | 62 (24.51) | 42 (26.58) | 40 (24.69) | 34 (23.29) | 36 (24.00) | 34 (23.94) |
aOther/unknown SMA type included those with a non-5q SMA
bTreatment includes any SMA DMT received through a clinical trial and/or commercially available therapy