2 results
Approved WMOCompleted
To evaluate the efficacy of rVWF (vonicog alfa) prophylaxis based on the annualized bleeding rate (ABR) of spontaneous (not related to trauma) bleeding episodes in adult and pediatric / adolescent subjects (aged 12 to <18 years) during the…
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.…