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 WMORecruiting
The primary objective to demonstrate that in CNOpatients with pain 3-monthly treatment with pamidronate will result in significant decrease in maximum pain score (as measured with BPI) as compared to placebo.Secondary objectives:•…