2 results
Approved WMOWill not start
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 theā¦
Approved WMORecruiting
The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in adult subjects with late-onset Pompe disease.