3 results
Primary Objective:*Evaluate the safety and tolerability of AMG 596 administered by continuous intravenous (cIV) infusion in monotherapy (Arm 1) and in combination with AMG 404 (anti-programmed cell death-1 (PD-1) antibody( Arm 2) in subjects with…
To test if tamoxifen treatment, compared to placebo, reduces the progression of the disease in 6.5-12 years old ambulant DMD patients by at least 50% (using the MFM D1 subscore as primary clinical endpoint in group A patients).To test if tamoxifen…
Primary:•To compare the effect of venglustat with standard of care Fabry therapies on left ventricular mass index over 18 months in participants with Fabry disease and left ventricular hypertrophySecondary:•To evaluate the effect of venglustat on…