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