4 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…
The overall objectives of the study are to evaluate the safety and efficacy (anatomical and functional visual outcomes) of two doses of GT005 in genetically defined subjects with GA due to AMD.
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 (…