Swedish Orphan Biovitrum

This study tests the drug anakinra in Chinese patients with two related inflammatory conditions: Systemic Juvenile Idiopathic Arthritis (SJIA) in children and Adult-Onset Still's Disease (AOSD) in adults. Participants receive a daily injection under the skin for 48 weeks. The goa…

This study tests whether anakinra (Kineret), a daily injection, can reduce monthly attacks in Chinese patients with Familial Mediterranean Fever (FMF) who do not respond to standard colchicine treatment. Three patients aged 2 years and older will receive anakinra for 6 months, wi…

This study tests whether a once-weekly medication (efanesoctocog alfa) can improve or prevent joint damage in 37 people with moderate or severe hemophilia A. Participants receive the drug for 12 months, and doctors use ultrasound and MRI to check joint health. The goal is to see …

This phase 3 study tests a drug called pacritinib against standard treatments chosen by the doctor in people with myelofibrosis who have very low platelet counts (below 50,000). The goal is to see if pacritinib can shrink the spleen and reduce symptoms like tiredness, pain, and i…

This 48-month study observes 427 people with hemophilia A who are already using Elocta to prevent bleeding. Researchers want to see how well the drug protects joints over time, including how many joint bleeds occur and whether target joints improve or worsen. The goal is to under…

This study was designed to observe how patients with hereditary tyrosinemia type 1 (HT-1) in China respond to nitisinone treatment in everyday medical practice. It planned to track serious health events like liver problems or death. However, the study was withdrawn before enrolli…