Swedish Orphan Biovitrum

This study follows 427 people with hemophilia A who are already taking Elocta to prevent bleeding. Over 48 months, researchers will track joint health, including bleeding episodes and joint damage. The goal is to see how well Elocta works in everyday life to protect joints.

This study tests a drug called pacritinib against standard treatments in about 400 people with myelofibrosis, a type of bone marrow cancer, who also have very low platelet counts (below 50,000). The goal is to see if pacritinib can shrink the spleen and improve symptoms like bell…

This study tests whether a once-weekly injection of efanesoctocog alfa can improve joint health in 37 people with moderate or severe hemophilia A. The main goal is to see if the drug reduces thickening of the joint lining (synovial hypertrophy) and prevents joint bleeds over 12 m…

This study is testing a new medicine called efanesoctocog alfa in about 90 people aged 12 and older with severe hemophilia A. Participants receive a weekly injection for 24 months. The goal is to see if this treatment helps them be more physically active and improves joint health…

This study was designed to observe patients with hereditary tyrosinemia type 1 (HT-1) in China who are already taking nitisinone. The goal was to track side effects, deaths, liver transplants, and liver cancer. However, the study was withdrawn before enrolling anyone, so no resul…