Table 1 Patient characteristics and follow-up*
Characteristics | n = 283 |
|---|---|
Female, n (%) | 149 (53) |
Median age at study entry, years (range) | 48 (19–81) |
Median age at diagnosis, years (range) | 38 (1–72) |
Median disease duration at study entry, years (range) | 9 (0–32) |
Country of residence, n (%) | |
Netherlands | 109 (39) |
United Kingdom | 23 (8) |
United States | 71 (25) |
Germany | 48 (17) |
Other† | 32 (11) |
Disease severity at study entry, n (%) | |
No wheelchair use or respiratory support‡ | 134 (47) |
Wheelchair use | 37 (13) |
Use of respiratory support | 42 (15) |
Both wheelchair use and respiratory support | 70 (25) |
Median follow-up time, years (range) | 6 (0.04-9) |
ERT during the course of the study, n (%) | 204 (72) |
Median ERT duration, years (range) | 4 (0.2-8) |
Median age at start of ERT, years (range) | 51 (24–76) |
Died during follow-up, n (%) | 46 (16) |
Median age at death, years (range) | 59 (23–86) |