3 results
the objective of the current study is to evaluate the safety and efficacy of enzyme therapy in the Dutch population of patients with MPS I, II and VI; to compare this to the natural course of these diseases; to make an inventory of the direct and…
Primary Objective:• To compare the progression-free survival (PFS) of patients randomized to mirvetuximab soravtansine (MIRV) vs. Investigator*s choice of chemotherapy (IC Chemo) Key Secondary Objectives:• To compare the objective response rate (ORR…
See protocol P5:The primary objective of this study is to evaluate the efficacy of 12 months of oral ACH-0144471 in participants with C3G or ICMPGNbased on histologic scoring and proteinuriaThe secondary objectives of this study are: * To evaluate…