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 overall objective is to assess the effectiveness of treating patients with corticosteroids prior to surveillance in order to diminish the pitfall of active inflammation.