2 results
Approved WMORecruiting
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…
Approved WMOCompleted
Primary Core: to demonstrate the efficacy of BAF312 relative to placebo in delaying the time to 3-month confirmed disability progression, measured by EDSS. Main secondary objectives Core: to demonstrate the efficacy in delaying the time to 3-month…