TABLE 1b.
Impact of individual symptoms and perceived effectiveness of available treatment options: Musculoskeletal and eye problems.
| Impact | Worry | Treatment option | Ideal treatment | ||||
|---|---|---|---|---|---|---|---|
| Age [years] | Symptom | Very high/most important | Very high/most important | Used | Effective | Very/most important | |
| Hypotonia/weak muscles1 | 0–3 | 92.3% | 76.9% | 42.3% | N/A | N/A | 73.1% |
| 4–10 | 84.4% | 53.1% | 34.4% | N/A | N/A | 75.0% | |
| 11–18 | 94.1% | 41.2% | 35.3% | N/A | N/A | 64.7% | |
| >18 | 83.3% | 33.3% | 66.7% | N/A | N/A | 83.3% | |
| Contracturesa | 0–3 | 84.6% | 30.8% | 19.2% | 57.7% | 57.7% | N/A |
| 4–10 | 71.9% | 31.3% | 21.9% | 59.4% | 56.3% | N/A | |
| 11–18 | 82.4% | 5.9% | 5.9% | 52.9% | 52.9% | N/A | |
| >18 | 83.3% | 16.7% | 33.3% | 33.3% | 33.3% | N/A | |
| Mobility problems/difficulty walkingb | 0–3 | 84.6% | 73.1% | 73.1% | 100.0% | 100.0% | 88.5% |
| 4–10 | 75.0% | 56.3% | 56.3% | 90.6% | 90.6% | 75.0% | |
| 11–18 | 94.1% | 47.1% | 52.9% | 100.0% | 100.0% | 82.4% | |
| >18 | 83.3% | 83.3% | 83.3% | 83.3% | 83.3% | 100.0% | |
| Scoliosisc | 0–3 | 23.1% | 42.3% | 30.8% | 11.5% | 11.5% | N/A |
| 4–10 | 34.4% | 28.1% | 25.0% | 18.8% | 18.8% | N/A | |
| 11–18 | 64.7% | 47.1% | 47.1% | 41.2% | 29.4% | N/A | |
| >18 | 83.3% | 83.3% | 83.3% | 16.7% | 16.7% | N/A | |
| Eye problems | 0–3 | 76.9% | 42.3% | 30.8% | N/A | N/A | N/A |
| 4–10 | 71.9% | 21.9% | 28.1% | N/A | N/A | N/A | |
| 11–18 | 82.4% | 23.5% | 17.6% | N/A | N/A | N/A | |
| >18 | 50.0% | 33.3% | 0.0% | N/A | N/A | N/A | |
Note: Muscular problems become less important whereas scoliosis causes high impact in older cohorts. n (0–3 years) = 26, n (4–10 years) = 32, n (11–18 years) = 17, n (>18 years) = 6. Therapeutic options: asplints and braces, bphysical therapy, cscoliosis brace; ideal treatment: 1improves stamina and activity.
Abbreviation: N/A, not asked.