3 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 objective of the study is demonstrate the safety and efficacy of the subcutaneous defibrillation system.
Approved WMOWill not start
The purpose of this study is to assess the safety, tolerability and efficacy of long term drisapersen in subjects with DMD.