Researchers sought to determine whether there is a link between recombinant factor VIII Fc fusion protein and time to tolerization in patients with severe hemophilia A .
Researchers sought to determine whether a lower bleeding rate would be achieved with prophylaxis with fitusiran for hemophilia A or B.
Researchers sought to develop recommendations for future research opportunities to address the priorities of patients with ultra-rare bleeding disorders.
Reduction in annualized bleeding rate seen compared with bypassing agents on demand and on-demand clotting factor concentrates.
Researchers sought to determine whether there were changes in participant-reported physical activity among people with moderate to severe hemophilia during the COVID-19 lockdown.
Researchers sought to determine whether TQG202 would lead to effective bleeding control when used on-demand for patients with moderate-to-severe hemophilia A.
Researchers sought to determine whether rondaptivon pegol would be an effective treatment to increase endogenous FVIII levels in patients with hemophilia A.
Researchers sought to determine whether inhibitor incidence with N8-GP prophylaxis would be within range for patients with previously untreated hemophilia A.
Researchers sought to determine whether romiplostim would be effective in treating pediatric patients with immune thrombocytopenia.
Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy.
