Swedish Orphan Biovitrum

This study tests 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, whi…

This study tests whether a once-weekly medicine called efanesoctocog alfa can improve joint health in 37 people with moderate or severe hemophilia A. Researchers will use ultrasound and MRI to check for joint swelling and damage over 12 months. The goal is to see if the treatment…

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 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 was designed to collect information on patients with hereditary tyrosinemia type 1 (HT-1) in China who are already taking nitisinone. The goal was to track side effects, deaths, and need for liver transplants during routine care. However, the study was withdrawn before…