Table 1 Patient characteristics
Patient characteristics | Late infantile (N = 13) | Early juvenile (N = 6) | Late juvenile (N = 2) | Adult onset (N = 3) |
|---|---|---|---|---|
Patients receiving no disease modifying treatment | 10 | 3 | 2 | 2 |
 Alive, n | 8 | 3 | 2 | 1 |
 Deceased, n | 2 | 0 | 0 | 1 |
 Age now | 5.6 (2–7) | 13.3 (11–16) | 24.0 (15–33) | 30 |
 Age at death | 6.3 (5–7.5) | - | - | 39 |
Patients receiving gene therapy or HSCTa | 3 | 3 | 0 | 1 |
 Alive, n | 3 | 3 | 0 | 1 |
 Deceased, n | 0 | 0 | 0 | 0 |
 Age now | 4.7 (4–5) | 11.3 (9–13) | - | 48 |
 Age at death | - | - | - | - |