Swedish Orphan Biovitrum

This study tested a drug called pegcetacoplan in 24 adults with cold agglutinin disease, a rare condition where the immune system destroys red blood cells. The goal was to see if the drug could raise hemoglobin levels and reduce the need for blood transfusions. Participants recei…

This study looked at how safe and effective emapalumab is for Chinese patients with primary hemophagocytic lymphohistiocytosis (pHLH), a rare and serious immune disorder. Thirteen patients who had already received treatment were given emapalumab. The main goal was to see how many…

This study followed 151 people with hemophilia B for 24 months to see how well Alprolix works in real-world settings. Researchers tracked bleeding episodes, how often the medicine was used, and the amount needed. The goal was to understand the drug's effectiveness and usage patte…

This study tested a new medicine called SEL-212 in 112 adults with chronic gout that standard treatments couldn't control. Participants received one of two doses of SEL-212 or a placebo every 28 days for about 6 months. The main goal was to see if SEL-212 could lower and keep uri…

This study tested a drug called pegcetacoplan in 12 adults who developed a serious blood clotting condition (TA-TMA) after a stem cell transplant. The goal was to see how the drug moves through the body, if it helps control the disease, and if it is safe. Participants received th…

This study tested a new medicine called SEL-212 in 153 adults with chronic gout that did not get better with standard treatments. The goal was to see if SEL-212 could lower uric acid levels in the blood to a safe range and keep them there for at least 80% of the time over six mon…

This study tested a drug called emapalumab in 33 children and adults with a life-threatening immune overreaction (macrophage activation syndrome) linked to Still's disease or lupus. The goal was to see if the drug could safely calm the immune system and improve blood markers and …

This study looked back at the medical records of 32 people with Still's disease who also developed a serious complication called macrophage activation syndrome (MAS). The goal was to understand their symptoms, how they were treated, and how they recovered. By analyzing past cases…