2 results
Approved WMOCompleted
Primary:• To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in ambulant boys ages 4 to <8 years with DMD using the Time to Stand Test (…
Approved WMOCompleted
The primary objective of the RAPID study is to determine whether etripamil nasal spray (NS) self-administered by patients is superior to placebo at terminating episodes of PSVT in an at-home setting.The secondary objective of this study is to…