3 results
The main purpose of this trial is to find out if an investigational drug called setmelanotide (RM-493), which mimics MSH, can help control body weight. The efficacy of setmelanotide will be compared to the efficacy of a placebo during 4 to 8 weeks.
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…
Currently, there are no curative treatment options for patients with Waldenstrom*s macroglobulinemia (WM) and there is no consensus on standard first line or relapse treatment. The proteasome inhibitor bortezomib has shown efficacy in WM, but is…