Swedish Orphan Biovitrum

This study is checking if a weekly preventative treatment (efanesoctocog alfa) can improve the health of joints already affected by swelling (synovial hypertrophy) and prevent new joint bleeds in people with moderate or severe hemophilia A. About 37 participants aged 12 and older…

This study is observing 427 people with hemophilia A over four years to see if long-term treatment with Elocta helps protect their joints from damage. Participants are already using Elocta as part of their regular care. The main goal is to see if this treatment reduces joint blee…

This study is looking at whether a weekly preventative treatment called efanesoctocog alfa can help people with severe hemophilia A become more physically active and improve the health of their joints over two years. About 90 patients aged 12 and older will use an activity tracke…

This study aimed to collect information on how children in China with a rare, serious liver disease (hereditary tyrosinemia type 1) fare while taking the medication nitisinone during their normal doctor visits. It was an observational study, meaning it would not test a new treatm…