Patients continuously treated with miglustat (N = 92) | Patients with age at neurological onset data (n = 84) | ||||
---|---|---|---|---|---|
EI (<2 years) | LI (2 – <6 years) | JUV (6 – <15 years) | AA (≥15 years) | ||
Male: female, n (%) | 47 (51.1): 45 (48.9) | 5 (55.6): 4 (44.4) | 12 (44.4): 15 (55.6) | 14 (46.7): 16 (53.3) | 10 (55.6): 8 (44.4) |
Age at onset of neurological manifestations | |||||
n | 84* | 9 | 27 | 30 | 18 |
Mean (SD), years | 9.8 (8.6) | 0.9 (0.7) | 4.2 (1.3) | 9.8 (2.7) | 22.7 (8.8) |
Median (range), years | 6.9 (<1–44.6) | 1.0 (<1 – 2.0) | 4.5 (2.0 – 5.9) | 9.2 (6.1 – 14.6) | 18.7 (15.5 – 44.6) |
Age at diagnosis | |||||
n | 87‡ | 9 | 27 | 28# | 17† |
Mean (SD), years | 13.7 (10.9) | 1.9 (1.9) | 7.8 (7.0) | 16.6 (7.7) | 26.7 (9.9) |
Median (range), years | 12.0 (0.1–44.7) | 1.4 (0.1 – 5.9) | 5.2 (0.1 – 28.2)** | 13.7 (7.7 – 40.2) | 22.1 (14.4 – 44.7) |
Age at enrolment | |||||
n | 92 | 9 | 27 | 30 | 18 |
Mean (SD), years | 18.9 (11.3) | 4.7 (2.3) | 12.5 (7.2) | 20.7 (8.3) | 31.2 (9.2) |
Median (range), years | 18.8 (0.7–48.3) | 4.1 (2.2–8.6) | 11.1 (3.6–29.4) | 20.7 (9.2–41.5) | 29.0 (18.7–48.3) |
Composite disability score at enrolment | |||||
n | 87†† | 9 | 26α | 29α | 18 |
Mean (SD) | 0.37 (0.23) | 0.43 (0.33) | 0.32 (0.24) | 0.45 (0.22) | 0.35 (0.17) |
Median (range) | 0.35 (0–1.00) | 0.52 (0 – 1.00) | 0.28 (0 – 1.00) | 0.44 (0 – 0.88) | 0.29 (0.06 – 0.81) |
Composite disability score at last follow up | |||||
n | 90β | 9 | 27 | 30 | 17 |
Mean (SD) | 0.44 (0.27) | 0.59 (0.36) | 0.46 (0.29) | 0.49 (0.24) | 0.31 (0.22) |
Median (range) | 0.36 (0–1.00) | 0.58 (0.06–1.00) | 0.38 (0.06–1.00) | 0.51 (0.00–0.94) | 0.29 (0.00–0.88) |