4 results
The general aim of this project is the primary prevention of the development of AS in patients with IBP, additional (SpA) features and inflammation on MRI of the SI joint and/or spine (but still no sacroiliitis on X-ray) by giving them, a short…
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 (…
The primary objective of Arm 1 is to evaluate the overall response rate (ORR) as determined by an independent radiology review committee (IRC) and measured by the proportion of patients with best overall confirmed response of complete response (CR)…