3 results
Approved WMOCompleted
Primary Objective: To study which test or which combination of tests has the best predictive value at baseline level for a positive effect of the ITB test-infusion, rated by the patient on the Patient-Global Impression of Change (P-GIC). Secondary…
Approved WMOCompleted
Please refer to protocol, section 1.2 "Rationale"
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.