3 results
Approved WMOCompleted
The aim of this project is to validate the hypothesis that intranasal insulin improves development and behaviour in children with Phelan-McDermid syndrome.
Approved WMOCompleted
Evaluate the long-term safety and effectiveness of two dose levels of orally administered BCX7353 in subjects with HAE
Approved WMOPending
Primary objective:- To evaluate the efficacy of caplacizumab in combination with immunosuppressive therapy (IST) without therapeutic plasma exchange (TPE) inadults with immune mediated thrombotic thrombocytopenic purpura (iTTP)Secondary objectives:…