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Table 1 Patient Characteristics of 268 untreated adult patients with Pompe disease

From: Survival and associated factors in 268 adults with Pompe disease prior to treatment with enzyme replacement therapy

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)
  1. 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).