3 results
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 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 (…
Primary:- To assess the safety and tolerability of MK2140- To evaluate objective response rate (ORR) of MK2140 as assessed by BICR per RECIST 1.1Secondary:-To evaluate the duration of response (DOR) of MK2140 as assessed by BICR per RECIST 1.1