Table 1.

Patient Characteristics of 268 untreated adult patients with Pompe disease

Female, n (%)	141 (53)
Median age at study entry, years (range)	48 (19-79)
Median age at diagnosis, years (range)	38 (1-68)
Number of patients diagnosed in age categories of 15 years, n (%)
<15 years	22 (8)
16-30 years	59 (22)
31-45 years	115 (43)
46-60 years	61 (23)
>61 years	11 (4)
Nationality, n (%)
Netherlands	99 (37)
Germany	48 (18)
US	69 (26)
UK	20 (8)
Australia	13 (5)
Canada	9 (3)
Other	10 (4)
Median disease duration at entry, years (range)	9 (0-32)
Median follow up time, years (range)	3.5 (0.02-7)
Disability level at study entry, n (%)
No wheelchair use or respiratory supportª	127 (47)
Wheelchair use	34 (13)
Use of respiratory support	39 (15)
Both wheelchair use and respiratory support	68 (25)
Median RHS score* at study entry (range) (n = 258)	27 (9-36)

Continuous variables are expressed as median (range). Categorical variables are expressed as n (%). ª'Respiratory support' includes partial and fulltime, invasive and non-invasive respiratory support *RHS assesses the level of participation/handicap; score varies between 9 (severe participation restrictions) and 36 (no participation restrictions).