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Table 3 Changes of secondary efficacy variables from baseline to 24 weeks

From: Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter Syndrome)

  Comparator 0.5 mg/kg/week (N) Idursulfase beta 0.5 mg/kg/week (N) Idursulfase beta 1.0 mg/kg/week (N)
6MWT, m -9.1±35.2 (8) 61.6±32.2 (6) 38.4±36.4 (7)
6MWT,% change -2.7±9.2 (8) +23.5±16.9 (6) +12.7±11.9 (7)
FVC, L 0±0.1 (5) 0±0.1 (3) 0.2±0.1 (4)
FVC,% change +1.2±8.4 (5) +7.9±11.3 (3) +15.8±7 (4)
LVMI,% change -1.7±18 (10) -5.1±18.5 (10) -5.9±25.1 (10)
LVEF,% change +2.9±11.9 (9) +2.5±10.8 (10) -1.4±7.4 (10)
Joint range of motion,% changes
Shoulder flexion 23.4±26.6 (10) 6.1±10.7 (10) 6.7±11.2 (10)
Shoulder extension 1.8±43 (10) -20.4±31.8 (10) -1.2±19.8(10)
Elbow flexion 7.2±10.7 (10) 1.1±9.4 (10) -1.6±11 (10)
Elbow extension 25±78.8 (9) 3.8±41.6 (10) 11.9±94.5 (10)
Hip flexion 4.5±14 (10) -4.1±14.9 (10) -1.3±10.9 (10)
Hip extension 22.6±41.5 (10) 47.3±98.9 (10) 4.2±34.2 (8)
Knee flexion 4.5±7.9 (10) -0.7±12.5 (10) 1.3±8.2 (10)
Knee extension 7.4±40.1 (9) -28.1±59.1(10) 0±43.6 (8)
  1. 6MWT, 6 minute walk test.
  2. FVC, functional vital capacity.
  3. LVMI, left ventricular mass index.
  4. LVEF, left ventricular ejection fraction.