Table 1.
Characteristics of untreated MPS I registrants with usable length/height measurements
| Severe MPS I | Attenuated MPS I | |||
|---|---|---|---|---|
| Male | Female | Male | Female | |
| Registrants with usablea initial measurements (n) | 463 | 207 | ||
| 236 | 227 | 94 | 113 | |
| Number of usable records (n) | 546 | 547 | 338 | 383 |
| Natural history follow‐up time (years) (median [IQR]) | 1.4 (1.0,2.5) | 1.4 (1.1,2.6) | 9.1 (4.6,15.6) | 9.4 (5.6,13.6) |
| Reported age at diagnosis (years) (median [IQR]) | 0.9 (0.6,1.3) | 1.0 (0.7,1.5) | 4.5 (3.2,6.6) | 4.8 (2.3,8.3) |
| Registrant distribution | ||||
| North America (n [%]) | 119 (50.4) | 125 (55.1) | 50 (53.2) | 46 (40.7) |
| Europe (n [%]) | 108 (45.8) | 89 (39.2) | 37 (39.4) | 59 (52.2) |
| Latin America (n [%]) | 5 (2.1) | 12 (5.3) | 7 (7.4) | 6 (5.3) |
| Asia‐Pacific (n [%]) | <5 | <5 | <5 | <5 |
Note: IQR = interquartile range (25%, 75%).
Abbreviations: IQR, interquartile range; MPS I, Mucopolysaccharidosis Type I.
a
Defined as data with units and that were physiologically plausible.