Swedish Orphan Biovitrum

This study is comparing how long a new hemophilia A treatment (efanesoctocog alfa) stays active in the bloodstream versus two existing treatments. It involves 24 adult men with severe hemophilia A who are already using one of the comparison treatments. Each participant will recei…

This study aims to understand how well a preventive treatment called efanesoctocog alfa protects joint health over time in people with hemophilia A. It will follow about 250 people already prescribed this medication in real-world settings for 2-3 years, tracking their joint bleed…

This study is testing if a drug called pacritinib can help control painful flare-ups in people with VEXAS syndrome, a rare and serious inflammatory blood disorder. The main goal is to see if the drug allows patients to safely reduce their steroid medication. 78 participants will …

This study is testing an investigational drug called pacritinib against standard care treatments for adults with myelofibrosis, a serious bone marrow cancer, who also have dangerously low platelet counts. The main goals are to see if pacritinib can reduce spleen size and ease dis…

This study allows patients with severe hemophilia A who benefited from a previous trial to continue receiving an experimental clotting factor treatment. It aims to provide ongoing treatment access while monitoring safety and effectiveness until the medication becomes commercially…