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Heme degradation and oxidative stress in murine models for hemoglobinopathies: thalassemia, sickle cell disease and hemoglobin C disease
Mid-range doses (10-20mg) showed optimal efficacy-to-safety ratio Phase III: ~500 subjects, 24 weeks, failed to reach primary endpoint (statistically significant weight loss vs placebo at 24 weeks)
Biomarker cut-off values were determined using Youdens index
