3 results
To demonstrate the feasibility to induce an effective, predictable and sustained decrease in FGF23 level in CKD stage I-IV patients, without inducing hypophosphatemia using a stepped treatment regimen aiming at restricting phosphate uptake.
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 (…