4 results
An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia A.…
Objectives The main objective of the FAIR-PARK II trial is to demonstrate an effect of DFP on the course of PD (including both disease-modifying and symptomatic effects). The trial's overall objective can be summarized as follows: to…
Studying the effect of deferiprone on the motor function of patients with Pelizaeus-Merzbacher disease.
The purpose of this study is to investigate the safety and efficacy of rFVIIIFc in previously untreated patients (PUPs) in accordance with the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) guideline on the…