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
The primary objective is to show a difference in responder rates between the study product (T4020) and the vehicle : a reduction of 50% or more in keratitis/ulcer area from baseline (inclusion visit = V2 = Day 0) assessed at Day 28 (Visit 6).