HEMOPHILIA A

This study tested a drug called emicizumab in 85 people with hemophilia A, a condition where blood doesn't clot properly. Participants received either weekly or monthly shots of emicizumab or no preventive treatment. The goal was to see if the drug could reduce the number of blee…

This study tested the real-world use of emicizumab in 71 infants and children aged 0-3 years with hemophilia A, a bleeding disorder. The goal was to see how many children could use the drug to prevent bleeding and how often breakthrough bleeds occurred. Results help doctors under…

This study tested a new gene therapy (SB-525) in 13 adults with severe hemophilia A, a bleeding disorder. The goal was to see if a single infusion could safely raise the body's own clotting factor levels and reduce bleeding episodes. Participants were monitored for up to 5 years …

This study tested a medicine called emicizumab in 73 people with mild or moderate hemophilia A (a bleeding disorder) who do not have inhibitors. The goal was to see if it safely reduces the number of bleeding episodes. Participants received regular injections, and researchers tra…

This study tested a new medicine called BIVV001 in 74 children under 12 with severe hemophilia A, a condition where blood doesn't clot properly. The goal was to see if the drug is safe and can help prevent or control bleeding episodes. The children received regular infusions of t…

This study followed 230 people with hemophilia A who were already prescribed Kovaltry to see how safe it is and how well it controls bleeding in everyday medical practice. Researchers tracked side effects and how many bleeds occurred. The goal was to gather real-world information…

This study tested a one-time gene therapy (valoctocogene roxaparvovec) combined with steroids in 22 men with severe hemophilia A. The goal was to raise their low clotting factor levels and reduce the need for regular factor infusions. Results showed increased factor activity and …

This study tested a new scoring method to help doctors choose the right dose of Jivi (a longer-lasting clotting factor) for people with hemophilia A. 21 participants switched from standard treatments to Jivi, and their dose plan was based on their personal risk score. The goal wa…

This study looked at the safety of BAY 94-9027 (Jivi) in 36 children aged 7 to 12 with severe hemophilia A. The drug is a longer-lasting clotting factor that requires fewer injections. Researchers monitored for allergic reactions and whether the drug stopped working well during t…

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 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 drugs. Participants received a single injection of each drug in a fixed order, and researche…

This study tracked 212 adults with moderately severe to severe hemophilia A or B for at least 6 months to record how often they bleed while on their usual clotting factor treatment. The information will be used as a baseline for future gene therapy studies. No experimental treatm…