2 results
Approved WMOPending
The aim of this pilot study is;* To determine whether differences between individuals in the set-point of the HPT-axis can be determined by using this study-design,* To examine whether this study design is practicable and tolerated by the healthy…
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…