3 results
The purpose of this study is to obtain blood samples from up to 14 subjects who previously received namilumab in the previous study M1-1188-002-EM (PRIORA, [NCT01317797]) to correlate genetic markers with clinical outcomes.
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…
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…