Swedish Orphan Biovitrum

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 checking if a weekly preventative treatment called efanesoctocog alfa can improve joint health in people with moderate or severe Hemophilia A. It will follow about 37 patients in Europe for 12 months, using ultrasound and MRI scans to see if the treatment reduces jo…

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…