2 results
Approved WMOCompleted
The aim of this study is the efficacy of a simplified triple regimen at an energy setting of 12 J/cm2 instead of 2x2x15 J/cm2, without a cleaning phase in between, and its effect on the rate of stenosis and overall complications when using the Barrx…
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…