Swedish Orphan Biovitrum

This study tests a drug called pacritinib for people with VEXAS syndrome, a rare genetic condition that causes severe inflammation. About 78 adults will receive either one of two doses of pacritinib or a placebo to see if it helps control symptoms. The goal is to find the best do…

This study follows 250 people with hemophilia A who are taking efanesoctocog alfa to prevent bleeding. The goal is to see how well the drug protects joints from bleeds over the long term in everyday life. Participants will have their joint bleed rates tracked for several years.

This study gives people with moderate or severe hemophilia A, aged 6 and older, continued access to the treatment efanesoctocog alfa after they finish a previous trial. Researchers will track how well the drug controls bleeding and monitor for any side effects. The goal is to pro…

This study compares a new clotting factor medicine (efanesoctocog alfa) with two existing ones in adults with severe hemophilia A. About 24 men aged 18-65 will receive a single dose of their current treatment and then a single dose of the new medicine. The main goal is to see how…