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Table 1 Patient demographics and baseline characteristics of patients ≥18 years of age in MOR-004/005 and MorCAP

From: Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa

  ITT (n = 37) MPP (n = 32) MorCAP (n = 10)a
Demographics
 Age, mean (SD), yrs 30.9 (9.7) 30.3 (8.5) 26.9 (7.8)
 Male, n (%) 13 (35.1) 12 (37.5) 1 (10.0)
 Ethnicity, n (%)
  White 30 (81.1) 26 (81.3) 8 (80.0)
  Nonwhite 7 (18.9) 6 (18.8) 2 (20.0)
 Height, mean (SD), cm 114.1 (20.4) 114.6 (20.8) 106.4 (13.1)
Baseline characteristics, mean (SD)
 6MWT distance, m 176.9 (67.3) 175.3 (70.7) 148.6 (109.1)
 3MSCT, stairs/min 21.3 (13.1) 22.6 (13.2) 16.6 (15.3)
 uKS, μg/mg 9.5 (6.2) 9.6 (6.5) 7.2 (5.0)
 FVC, L 1.5 (1.1) 1.5 (1.1) 0.9 (0.3)
 FEV1, L 1.2 (0.8) 1.3 (0.9) 0.7 (0.3)
 MVV, L/min 43.2 (31.7) 44.9 (33.9) 29.8 (15.2)
 HAQ—self-care 2.7 (2.2) 2.0 (1.9) 2.4 (1.9)
 HAQ—caregiver assistance 23.0 (10.0) 23.7 (10.5) 25.8 (8.0)
 HAQ—mobility 5.4 (2.7) 5.5 (2.5) 5.4 (2.4)
  1. 3MSCT 3-min stair climb test, 6MWT 6-min walk test, FEV 1 forced expiratory volume in 1 s, FVC forced vital capacity, HAQ Health Assessment Questionnaire, ITT intent to treat, MPP modified per-protocol, MVV maximal voluntary ventilation, uKS urinary keratan sulfate
  2. a n = 9 for 6MWT, 3MSCT, FVC, and FEV1; n = 7 for MVV; n = 4 for uKS. Includes only patients with data at year 2