HEMOPHILIA A

This study looked at how well the drug emicizumab works in infants and children up to 3 years old with hemophilia A, a bleeding disorder. Researchers tracked how many children had breakthrough bleeding while on the drug and whether they needed extra clotting factor. The goal was …

This study tested a gene therapy called BAX 888 in 4 adult men with severe hemophilia A, a bleeding disorder. Participants received a single intravenous infusion of the therapy to help their bodies produce a missing clotting protein. The main goals were to check for side effects …

This study tested a new scoring method to help doctors choose the best Jivi treatment plan for people with hemophilia A. 21 participants switched from standard factor VIII products to Jivi, and their dose frequency was tailored based on their risk of bleeding. The goal was to see…

This study tested a medicine called emicizumab in 73 people with mild or moderate hemophilia A who do not have inhibitors. The goal was to see if it safely reduces bleeding episodes. Participants received regular injections, and researchers tracked how many bleeds occurred. The r…

This study tested a gene therapy called SB-525 in 13 adults with severe hemophilia A. The goal was to see if a single infusion could help their bodies produce the missing clotting factor, reducing or eliminating the need for regular factor injections. Participants were followed f…

This study tested a new medicine called BIVV001 in 74 children under 12 with severe hemophilia A, a condition where blood does not clot properly. The goal was to see if the drug is safe and can prevent bleeding episodes when given regularly. The drug is a lab-made clotting factor…

This study tested a one-time gene therapy (valoctocogene roxaparvovec) in 22 adults with severe hemophilia A. The goal was to help their bodies produce their own clotting factor VIII, reducing or stopping the need for regular factor infusions. Participants also received steroids …

This study tested a medicine called emicizumab in 85 people with hemophilia A, a condition that causes frequent bleeding. Participants received either weekly or monthly injections of emicizumab, or no preventive treatment. The goal was to see if emicizumab could reduce the number…

This study looked at the safety of BAY 94-9027 (Jivi) in 36 children aged 7 to 12 with severe hemophilia A. The medicine is a longer-lasting clotting factor given as an injection to prevent and treat bleeding. Researchers monitored for allergic reactions and whether the drug stop…

This study gathered information on how often adults with moderate to severe hemophilia A or B experience bleeding while on their usual clotting factor treatment. The goal was to create a baseline to compare against future gene therapy results. About 212 participants took part, an…

This study tested a new drug called efanesoctocog alfa in 13 adults with severe hemophilia A. The goal was to see how long it stays active in the body compared to standard clotting factor treatments. Participants received single injections of each drug in a fixed order, and resea…

This study looked at how safe and effective Kovaltry is for people with hemophilia A when used in everyday medical practice. Researchers tracked side effects and how well the drug controlled bleeding episodes in 230 patients. The goal was to gather real-world information, not to …