4 results
Primary:Is the degree of pupildilation, achieved after administrating a 4.5 microliter microdrop tropicamide 0.5% equivalent to the pupildilation after a regular 30 microliter eydrop tropicamide 0.5% in healthy volunteers?Secondary: Is there a…
Primary Objective:• To evaluate the safety and tolerability of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg in boys who have completed the NS 065/NCNP 01-301 study.Secondary Objective:• To compare the efficacy of…
Primary:To assess the safety and tolerability of DS-1211b compared with placebo in subjects with PXE.To assess the dose response by assessing the treatment changes in PD endpoints.
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 (…