2 results
Approved WMORecruiting
Part 1: To evaluate the efficacy and safety of imetelstat in transfusion dependent subjects with low or intermediate-1 risk MDS that is relapsed/refractory to ESA treatment.Part 2: To compare the efficacy, in terms of red blood cell (RBC)…
Approved WMOCompleted
Primary objectives: 1. To compare the efficacy of vamorolone administered orally at daily doses of 6.0 mg/kg over a 24-week treatment period vs. placebo in ambulant boys ages 4 to <7 years with DMD; and2. To evaluate the safety and…