Table 1 Baseline characteristics of Pompe patients included in the cost-effectiveness analyses (n = 90)

Age at first visit

49.1 years

50.0 years

23.0–75.0 years

Disease duration (since diagnosis)

7.7 years

4.3 years

0.0–27.6 years

Female

48%

Residual Enzyme activity (in fibroblasts)

12.0%

12.0%

0.5–19.9%

Wheelchair use

31%

Ventilation use

27%

Period at risk in ST survival analyses

3.5 years

3.5 years

0.0–8.9 years

Period at risk in ERT survival analyses

3.4 years

3.7 years

0.2–8.4 years