2 results
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.
Approved WMOCompleted
The main objective is to determine if bisphosphonate therapy with etidronate leads to stabilization or attenuation of ongoing calcification in the leg arteries as quantified by 18F-sodium fluoride(18F-NaF) PET-CT imaging in patients with PXE.…