Table 3.
Clinical characteristics of paediatric patients with dystrophinopathies, spinal muscular atrophy and myotonic dystrophy
| Diagnosis (N) | Average age (years) | Clinical trial participation | Ambulation (N) | Non-invasive ventilation (N) | Invasive ventilation (N) | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|
| Yes (no, unknown) | Yes | No | Unknown | Yes | No | Unknown | Yes | No | Unknown | ||
| Dystrophinopathies (249) | 12 | 60 (148, 41) | 178 | 45 | 25 | 6 | 216 | 25 | 0 | 224 | 25 |
| Spinal muscular atrophy (85)* | 9 | 3 (49, 33) | 7 | 34 | 6 | 24 | 46 | 15 | 2 | 68 | 15 |
| Myotonic dystrophy (58) | 10 | No data | 39 | 12 | 7 | 6 | 43 | 9 | 3 | 46 | 9 |
*Only includes spinal muscular atrophy patients for whom medical information is available (i.e., indexed patients)